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Archive for the ‘Crispr’ Category

ETF of the Week: ARK Genomic Revolution Multi-Sector Fund (ARKG) – ETF Trends

ETF Trends CEO Tom Lydon discussed the ARK Genomic Revolution Multi-Sector Fund (ARKG)on this weeks ETF of the Week podcast with Chuck Jaffe on the MoneyLife Show.

The ARK Genomic Revolution ETF (ARKG) is an actively-managed fund from the team at ARK Invest that tries to pick the companies best positioned to profit from advancements in energy, automation, manufacturing, materials, and transportation.

ARKG is an outperforming biotech ETF. As far as 1-month performances: ARKG is +43.7% versus the benchmark Nasdaq Biotechnology Index at +26.4%. Year-to-date, ARKG is +15.7% versus Nasdaq Biotechnology Index at +2.4%.

Concerning today, there is short-term support from the hope of a viable COVID-19 drug. For example, Pluristem (PSTI) surged on news that it had treated seven severely ill patients with COVID-19 in Israel with its allogeneic placental expanded (PLX) cells, with a survival rate of 100%, and It has treated at least one patient in the United States as well.

ARKG capitalizes on innovative, specialized drugs that are being developed by many overlooked names. Iovance (IOVA) climbed after it announced it would present data on its Phase 1 Study Combining Tumor-Infiltrating Lymphocytes (TIL) and Nivolumab in Non-Small Cell Lung Cancer. Compugen (CGEN) jumped on news that the company will present potentially positive updates on its ongoing Phase 1 immuno-oncology clinical trial evaluating COM701.

Looking at the long-term outlook, ARKG includes companies that merge healthcare with technology and capitalize on the revolution in genomic sequencing. These companies try to understand better how biological information is collected, processed, and applied by reducing guesswork and enhancing precision, restructuring health care, agriculture, pharmaceuticals, and improving our quality of life.

The convergence of Artificial Intelligence (AI), Next Generation DNA Sequencing (NGS) and CRISPR gene-editing has the potential to boost the efficiency of drug development radically. Breakthroughs in genomic science can present new treatments to help patients recover from what were once believed to be incurable afflictions.

For the record, the global genomics market was worth $851.96 million in 2019. It is expected to grow at a compound annual growth rate (CAGR) of 14.71% and reach $1.5 billion by 2023. Rising government funds for research on genomics drives the growth of the single-cell genomics market. The government funding focuses on efforts to resolve the complexity of the human genome, the genomic basis of human health and disease, and ensure that genomics is used safely to enhance patient care and benefit society through government, public and private institutions.

Related:ETF of the Week: ProShares Long Online/Short Stores ETF (CLIX)

Scientists have identified more than 50,000 genetic diseases caused by single-gene mutations, many of which are likely to be treated through genomic approaches, including several methods that have already begun to receive FDA approval. Looking ahead, CRISPR-based innovations to accelerate given the technologys ease of use, cost-efficacy, a growing body of research surrounding its safety and AI-powered CRISPR nuclease selection tools. CRISPR could also be utilized to address some of the most prominent healthcare problems, which opens up a significant investment opportunity in monogenic diseases.

Bolstering the case for ARKG over the long-term is the importance of genomics in an array of clinical trials. Drug development companies are making clinical trials more efficient by using NGS to find and enroll patients likely to respond. Half of the clinical trials and 80% of oncology trials now collect genetic information. ARK believes that clinical trials using genetic diagnostics will result in fewer failed drugs and will increase capital efficiency.

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ETF of the Week: ARK Genomic Revolution Multi-Sector Fund (ARKG) - ETF Trends

Yield10 Bioscience Obtains Positive Response from USDA-APHIS on Regulatory Status of its CRISPR Genome-Edited C3007 Trait in Camelina, Paving the Way…

WOBURN, Mass., April 27, 2020 (GLOBE NEWSWIRE) -- Yield10 Bioscience, Inc. (Nasdaq:YTEN), an agricultural bioscience company, today announced that it has obtained a positive response from USDA-APHISs Biotechnology Regulatory Services (BRS) for its CRISPR genome-edited C3007 trait in Camelina sativa plant lines designed to increase oil content. Yield10s submission along with the USDA-APHIS BRS response is posted on theUSDAs website.

In January 2020, Yield10 submitted an Am I Regulated? letter to the BRS, requesting confirmation of the regulatory status for Camelina plant lines containing the Companys novel, CRISPR genome-edited C3007 trait. The positive USDA-APHIS response came in the form of a published letter indicating that the plant lines do not meet the definition of a regulated article under 7 CFR Part 340.

This clarification of the regulatory status under USDA-APHIS guidelines accelerates the path for Yield10 to conduct field trials of the CRISPR genome-edited C3007 plants in the United States in the 2020 growing season. The plant lines may still be subject to regulation by the U.S. Environmental Protection Agency (EPA) or the U.S. Food and Drug Administration (FDA).

Receiving a positive response from USDA-APHIS for our CRISPR genome-edited C3007 lines is a critical milestone within our development program and facilitates the transition of lines with this trait to field testing this year, said Dr. Kristi Snell, Ph.D., Chief Science Officer of Yield10 Bioscience. Initial studies with C3007 have demonstrated potential for increased oil content, and the next step of testing this trait under field conditions will help us to characterize its performance and role in boosting oil content in Camelina and other oilseed crops.

The ability to increase oil content in specialty oilseed crops like Camelina has the potential to make a significant impact in the supply of omega fatty acid containing oils, to human nutrition and aquaculture feed markets. Further, the continued analysis of C3007 and its role as a key regulator of oil content in Camelina may also enable this trait to be used to increase production of edible oils in other major oilseed crops such as soybean and canola.

Once again, we appreciate both the speed and the transparency in which USDA-APHIS reviewed our letter and the data we provided, which has enabled us to plan our first field trials with this trait in 2020, said Dr. Oliver Peoples, Chief Executive Officer of Yield10 Bioscience. It is this sound, science-based regulatory framework that we believe is so important to the successful development and commercialization of new technologies for agriculture to increase crop performance, as well as other efforts to address sustainable global food security.

Yield10 licensed C3007 from the University of Missouri (MU) in 2018. The protein encoded by C3007, also known as BADC, is a novel regulator of the enzyme acetyl-CoA carboxylase (ACCase), the key enzyme for producing fatty acids for oil biosynthesis. In pilot studies conducted by MU researchers, reducing activity of the protein encoded by C3007 resulted in significantly increased oil content in seeds.Yield10 researchers have successfully used CRISPR to inactivate a number of the C3007 gene copies in Camelina and have seen clear evidence of increased oil content in some lines in laboratory studies. The use of CRISPR to deploy the trait may enable an expedited timeline for development and commercialization within the U.S. market.

The CRISPR genome-edited C3007 trait could deliver significant economic value by changing the value equation for the commercialization of identity preserved, specialty oilseed crops where the key value-driver is oil content with improved nutritional profiles for human consumption or for aquaculture feed or industrial markets. These traits may also be used to increase production of edible oils in major oilseed crops such as soybean and canola.

AboutYield10 Bioscience

Yield10 Bioscience, Inc. is an agricultural bioscience company developing crop innovations to improve crop yields and enhance sustainable global food security. The Company utilizes its proprietary GRAIN (Gene Ranking Artificial Intelligence Network) gene discovery platform to identify gene targets to improve yield performance and value in major commercial food and feed crops. Yield10 uses its Camelina oilseed platform to rapidly evaluate and field test new trait leads enabling the translation of promising new traits into the major commercial crops. As a path toward commercialization, Yield10 is pursuing a partnering approach with agricultural companies to drive new traits into development in crops such as canola, soybean and corn. The Company is also developing Camelina as a platform crop for producing nutritional oils and specialty products such as PHA biomaterials for use in water treatment applications. Yield10 is headquartered in Woburn, MA and has an Oilseeds Center of Excellence in Saskatoon, Canada.

For more information about the company, please visitwww.yield10bio.com, or follow the Company onTwitter,FacebookandLinkedIn.

(YTEN-G)

Safe Harbor for Forward-Looking Statements

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical, including, without limitation, statements regarding the Company's ability to conduct field testing of C3007 in Camelina plant lines in 2020, the possibility that testing this trait under field conditions will help us to characterize the performance of the trait and its role in boosting oil content in Camelina and other oilseed crops, the ability of C3007 to increase oil content in Camelina and other oilseed crops, the potential to make a significant impact in the supply of omega fatty acid containing oils to human nutrition and aquaculture feed markets, the potential for the trait to be used to increase production of edible oils in other major oilseed crops such as soybean and canola, the potential for an expedited timeline for development and commercialization within the U.S. market, the possibility for the trait to deliver economic value in other areas, and the possibility of translating promising new traits into the major commercial crops, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including the risks and uncertainties detailed in Yield10 Bioscience's filings with the Securities and Exchange Commission. Yield10 assumes no obligation to update any forward-looking information contained in this press release or with respect to the matters described herein.

Contacts:Yield10 Bioscience:Lynne H. Brum, (617) 682-4693,LBrum@yield10bio.com

Investor Relations Contact:Bret Shapiro, (561) 479-8566,brets@coreir.comManaging Director, CORE IR

Media Inquiries:Eric Fischgrund,eric@fischtankpr.comFischTank Marketing and PR

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Yield10 Bioscience Obtains Positive Response from USDA-APHIS on Regulatory Status of its CRISPR Genome-Edited C3007 Trait in Camelina, Paving the Way...

Analysts Estimate CRISPR Therapeutics AG (CRSP) to Report a Decline in Earnings: What to Look Out for – Yahoo Finance

Wall Street expects a year-over-year decline in earnings on higher revenues when CRISPR Therapeutics AG (CRSP) reports results for the quarter ended March 2020. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.

The earnings report might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower.

While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise.

Zacks Consensus Estimate

This company is expected to post quarterly loss of $1.09 per share in its upcoming report, which represents a year-over-year change of -17.2%.

Revenues are expected to be $6.35 million, up 1824.2% from the year-ago quarter.

Estimate Revisions Trend

The consensus EPS estimate for the quarter has remained unchanged over the last 30 days. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period.

Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggregate change.

Price, Consensus and EPS Surprise

Earnings Whisper

Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. This insight is at the core of our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction).

The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier.

Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only.

A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP.

Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell).

How Have the Numbers Shaped Up for CRISPR Therapeutics AG?

For CRISPR Therapeutics AG, the Most Accurate Estimate is the same as the Zacks Consensus Estimate, suggesting that there are no recent analyst views which differ from what have been considered to derive the consensus estimate. This has resulted in an Earnings ESP of 0%.

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On the other hand, the stock currently carries a Zacks Rank of #2.

So, this combination makes it difficult to conclusively predict that CRISPR Therapeutics AG will beat the consensus EPS estimate.

Does Earnings Surprise History Hold Any Clue?

Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number.

For the last reported quarter, it was expected that CRISPR Therapeutics AG would post earnings of $0.04 per share when it actually produced earnings of $0.51, delivering a surprise of +1,175%.

Over the last four quarters, the company has beaten consensus EPS estimates two times.

Bottom Line

An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss.

That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported.

CRISPR Therapeutics AG doesn't appear a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

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Analysts Estimate CRISPR Therapeutics AG (CRSP) to Report a Decline in Earnings: What to Look Out for - Yahoo Finance

‘At home’ coronavirus test? How CRISPR could change the way we search for COVID-19 – Genetic Literacy Project

If we take the advice of health experts, we wont be attempting a return to normal life in the US until we get better at identifying people infected with the novel coronavirus. That need is driving researchers across the nation to look for ways to expand our toolbox of testing options. And now a new test, developed using CRISPR gene editing technology, has been added to the mix.

About 5.4 million tests have been done in the US, according to the COVID Tracking Project, in a population of 328.2 million. That might sound like enough to keep ahead of an infectious disease that has only killed in five figures, but such an assumption grossly oversimplifies the situation.

Controlling the pandemic in the US is going to require a daunting number of diagnostic tests not just for the sick, but to verify when theyre better (two tests 24 hours apart for hospital discharge), in contact tracing to limit spread, and in the many individuals whove been infected but have few or no symptoms.

In a few countries, the use of diagnostic testing on a massive scale has been a cornerstone of successful containment strategies, write Matthew P. Cheng, MDCM. McGill University Health Centre and colleagues in a recent article in Annals of Internal Medicine. The US isnt on that list and has been struggling to catch up.

Simply put, we need faster, cheaper and more accurate tests than what we currently have. If they can be administered at home or in informal settings, all the better. Among the potential solutions for this challenge are efforts to turn everyones favorite gene editing tool, CRISPR, against the coronavirus. If successful, we could see a shift in access to tests. Imagine being able to use a nasal swab that delivers a yes or no verdict on coronavirus infection in under 40 minutes essentially like a home pregnancy test.

A CRISPR-based diagnostic is in the sights of researchers at the University of California, San Francisco Medical School and Mammoth Biosciences, which recently revealed plans for an inexpensive, compact test.

I can run it now myself at home, said Dr. Charles Chiu, professor of laboratory medicine at UCSF and co-lead developer of the new test, in an interview with NPR. Still, he acknowledged the test, which is being submitted for FDA approval, isnt yet simple enough for the average person to operate. He expressed confidence that a home-based test for nonexperts is within reach.

What we really want to develop is something like a handheld, pocket-sized device using disposable cartridges, he told NPR.

So, where would such CRISPR-based tests fit within the universe of coronavirus diagnostics? Theres little reason to think they would replace other testing options. Rather, think of them as offering another option for hospitals, clinics, doctors, and even consumers. That said, lets take a look at where we are in testing.

Diagnostic tests use a version of the polymerase chain reaction (real-time reverse transcriptase PCR) to amplify pieces of the RNA genome of SARS-CoV-2, the virus behind COVID-19. Dr. Deborah Birx, Coronavirus Response Coordinator, suggested that antigen-based tests be added to increase the supply, but these detect protein antigens on the viral surface, not the genetic material, and may be less likely to indicate infectious virus. For influenza diagnostic testing antigen tests are less accurate than the PCR-based tests.

PCR is simple in concept, but not always easy to carry out. It requires raising and lowering the temperature repeatedly as nucleic acid sequences are copied. Reagents may run out, and it requires specialized equipment. A PCR-based test that could diagnose infection in a few hours often takes more than a day because the sample must be sent to a lab.

False negatives can occur if viral RNA in a sample degrades during shipping, or if not enough collects on the swab. If either happens and a patient worsens, another test is required. But if the virus descends into the lungs, not enough may remain in the throat for a test to pick it up, even though the patient is actually sicker.

And so some people with obvious symptoms of COVID-19 must have two or three tests before a result is positive.

But PCR-based tests are what we have. Regulations shifted into emergency mode to bolster supplies.

On February 4, the Food and Drug Administration (FDA) greenlighted the Emergency Use Authorization (EUA) of the Center for Disease Control and Preventions (CDC) PCR test, making it available to to state and local public health labs and the Department of Defense. The EUA enables rapid roll-out of a still-experimental test. Even though PCR has been used in clinical diagnostics for decades, the virus is new and therefore so is the test.

A PCR-based diagnostic for COVID-19 amplifies RNA sequences unique to the novel virus, as well as gene parts common to other coronaviruses and a control sequence (encoding an enzyme, RNase), to make sure the test is working.

But at the end of January, bits of viral RNA in the CDC facility tainted some of the first test kits, so that they could yield false positive results, delaying their use.

On February 28, FDA took further action, announcing that clinically-licensed labs can use in-house developed tests while awaiting the EUA. These labs are CLIA-compliant,which means that they satisfy the standards of the Clinical Laboratory Improvement Amendments set by the federal government.

So places like Meridian Health, the Cleveland Clinic, Stanford Medicine, and many others began to do part of the PCR work-up, so that they didnt have to outsource samples. It was a little like people addicted to going to Starbucks learning how to make their own brew at home maintaining standards but taking on more of the task. (In fact, a study was just published on how to make the healthiest at-home brew.)

Michelle N. Gong, MD, Chief of Critical Care Medicine at Montefiore Medical Center in the Bronx, said on a JAMA Network webinaron March 23 that bringing the testing in-house immediately escalated testing.

We started with sending samples to the Department of Health, but it became increasingly clear that it was not going to be adequate. It took days. Our hospitals epidemiologist worked to bring testing onsite and that has changed the game. The ability to test onsite and turn it around fast made it much more efficient to get patients what they need.

The FDAs list of diagnostics granted EUA status continues to grow. The last time I checked it had 62 entries.

On March 27, the EUA granted Abbott Labs use of a test that they had under development that detects two viral genes (N and RdRp), one of them different from the CDCs recipe. According to the company, the test can deliver positive results in 5 minutes and negative results in 13 minutes.

The Abbott test runs on an existing platform, ID NOW, and uses a gene amplification technology that doesnt require the temperature shifts of PCR. The test is done in a lightweight box about the size of a toaster thats already used in doctors offices, urgent care facilities, and emergency departments to rapidly diagnose influenza, strep, and respiratory syncytial virus.

But the companys initial forecast of providing 50,000 tests a day, starting soon, may need to await further validation. A researcher at the Cleveland Clinic, Gary Procop, MD, tested five products on 239 patient samples known to be positive for COVID-19. Abbotts test missed 15 percent.

The World Health Organization (WHO)also provided many tests early on and continues to do so.

On April 21, FDA re-deployed the Emergency Use Authorization of LabCorpsPCR-based test for use in a home-kit that the company provides. Its being rolled out for health care workers first; consumer tests may follow in a few weeks.

Another way to boost test kit supplies is to harness the gene editing tool CRISPR. It may be faster and simpler than PCR.

The team from Mammoth Biosciences and UCSF reported on CRISPRCas12-based detection of SARS-CoV-2 in April 16s Nature Biotechnology. Company co-founder is Jennifer Doudna, PhD, co-inventor of CRISPR.

Mammoths visual readout strip works at the bedside, is fast, and in trials so far, picks up 100% of negatives and 95% of positives although much more extensive evaluation is needed. The report considers findings in 36 patients with COVID-19 and 42 people with other respiratory illnesses.

On February 15 Mammoth unveiled the protocol for their point of care test in a white paper that describes it as useful in areas at greatest risk of transmitting SARS-CoV-2 infection, including airports, emergency departments, and local community hospitals, particularly in low-resource countries.

We need faster, more accessible and scalable diagnostics. The point-of-care testing space is ripe for disruption and CRISPR diagnostics have the potential to bring reliable testing to the most vulnerable environments, said Mammoths Chief Technology Officer Janice Chen, PhD.

The test uses the companys DETECTR technology; that stands for DNA Endonuclease-Targeted CRISPR Trans Reporter. The platform had already been in the works for human papillomavirus, described in a April 27, 2018 report in Science.

The company started revamping its system for the novel coronavirus as soon as cases were reported from Wuhan, and within two weeks was testing it on the first patient samples. Because CRISPR can be programmed to detect any DNA or RNA sequence, we reconfigured our DETECTR platform within days to detect the SARS-CoV-2 virus from one of the first confirmed cases in the U.S., said Dr. Chen.

The system collapses the two steps of PCR-based tests into one: copying viral RNA into DNA and amplifying it fast, without temperature shifts. The test snips off a reporter molecule that generates the stain on the paper read-out strip, and uses the Cas12enzyme, which makes more precise cuts than conventional Cas9.

The test zeroes in on three gene pieces:

It picks up 70 to 300 DNA pieces of genetic material per microliter of fluid from a nose or throat swab. A microliter is one-millionth of a liter.

SHERLOCK, for Specific High Sensitivity Enzymatic Reporter Unlocking, is a testing platform coming from work on Zika virus disease and Dengue fever published in 2018 in Science. Sherlock Biosciences provides the test and Cepheid.com provides their GeneXpert Systems cartridge device. The approach licenses work from the Broad Institute, home of another CRISPR founder, Feng Zhang, PhD.

The system can identify any genetic target, and therefore any infectious disease. The test for COVID-19 may reach the market as a dip stick, paper strip, or even an electrochemical readout that can be read with a mobile phone, according to company information.

The system zeroes in on genome pieces unique to SARS-CoV-2 that encode the spike (S) protein and apolyprotein that commandeers the host cell. (My blog post from the start of the pandemic, COVID-19 Vaccine Will Close in on the Spikes, explains the genetic make-up of the virus.)

It uses powerful Cas12 and Cas13 enzymes and can reportedly detect down to the single molecule level. Its fast, accurate, and works directly on body fluids. And with 23,000 GeneXpert Systems already at health care facilities, popping in a COVID test may be the best idea yet.

To biologists and many others it was clear from the start that fighting this pandemic would require far more testing than for just the people who show up at health care facilities with symptoms. An epidemic is a population phenomenon that must be addressed at that level. It is comforting to know that regulations were in place to allow implementation of variations on the testing theme, and that the companies that have long expected a viral pandemic are collaborating to rapidly adapt existing tests, tools and technologies to put the pandemic of 2020 behind us.

Ricki Lewis is the GLPs senior contributing writer focusing on gene therapy and gene editing. She has a PhD in genetics and is a genetic counselor, science writer and author of The Forever Fix: Gene Therapy and the Boy Who Saved It, the only popular book about gene therapy. BIO. Follow her at her website or Twitter @rickilewis

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'At home' coronavirus test? How CRISPR could change the way we search for COVID-19 - Genetic Literacy Project

Diabetes reversed in mice using CRISPR and stem cell therapy – BioNews

27 April 2020

Genome-edited human stem cells from diabetic patientshavebeen shown to successfully reverse diabetes in mice.

Researchers at Washington University School of Medicine in St Louis, Missouri, used CRISPR/Cas9 to correct a genetic defect in human stem cells and then converted them into cells capable of producing insulin. When these edited insulin-producing cells were implanted into diabetic mice, they were able to effectively control blood sugar levels for six months.

'We're excited about the fact that we were able to combine these two technologies - growing beta cells from induced pluripotent stemcells and using CRISPR to correct genetic defects,' said corresponding author Dr Jeffrey Millman. 'This is the first time CRISPR has been used to fix a patient's diabetes-causing genetic defect and successfully reverse diabetes.'

The human cells used were from a patient with a rare genetic type of diabetes called Wolfram syndrome, which develops during childhood and typically requires affected patients to inject insulin multiple times each day.

'For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping-stone toward applying gene therapy to a broader population of patients with diabetes,' said Dr Millman.

The same team previously developed a new technique to more efficiently convert human stem cells into insulin-producing cells, allowing them to 'functionally cure' diabetes in mice for the first time (see BioNews1037). In the current study, they went one step further, adding the genome editing step to make cells from a diabetic personproduce insulin.

'We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation,' said Dr Fumihiko Urano, who co-led the study. 'In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes,' added Dr Millman.

'It's also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration' said Dr Urano.

The study was published in the journal Science Translational Medicine.

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Diabetes reversed in mice using CRISPR and stem cell therapy - BioNews

The US already has the technology to test millions of people a day – MIT Technology Review

There is widespread agreement that the only way to safely reopen the economy is through a massive increase in testing. The US needs to test millions of people per day to effectively track and then contain the covid-19 pandemic.

This is a tall order. The country tested only around 210,000 people per day last week, and the pace is not increasing fast enough to get to millions quickly.

The urgency to do better is overwhelmingly bipartisan, with the most recent legislation adding $25 billion for testing a few days ago. Fears are growing, however, that testing might not scale in time to make a difference. As Senators Lamar Alexander and Roy Blunt wrote last week, We have been talking with experts across the government and the private sector to find anyone who believes that current technology can produce the tens of millions of tests necessary to put this virus behind us. Unfortunately, we have yet to find anyone to do so.

We believe that it can be done. The scientific community has the technological capabilities today to test everyone who needs it and enable people to come back to work safely.

To be clearthe senators are right that simply scaling up current practices for covid testing is insufficient. However, with a bit of innovation, the US can meet the need without inventing entirely new technologies. The necessary scale can be achieved by deploying the fruits of the last decade of innovation in biology, including the dizzying advances in DNA sequencing, genetic engineering, industrial automation, and advanced computation.

We speak from experience. We have worked with and helped engender many of these technologies across academia and industry. Scaling them for widespread testing will require investment, infrastructure, and determination, but nothing technologically or logistically infeasible.

Tests for mass screening may have different requirements and characteristics from the tests run in clinical labs today that are approved by the Food and Drug Administration. So what might a solution look like?

It must be scalable, meaning tens or hundreds of thousands of tests per day per facility, or at-home tests. It must be sensitive to early stages of infection, detecting the actual virus rather than immunity to it. And it must be less bound by health insurance and regulatory constraints, to allow fast and broad testing, contact tracing, and isolation. These differences do not mean lower standards. In fact, screening at this scale will require stringent requirements for safety, accuracy, and reliability.

The life sciences community is rising to the challenge. We are repurposing our labs to advance new centralized and at-home methods that solve the bottlenecks preventing testing from reaching global scale. This community is moving fast, with shared purpose and a commitment to open collaboration. As a result of these efforts, several promising avenues are emerging.

Some rely on DNA sequencing tools that have improved a million-fold since the completion of the Human Genome Project nearly 20 years ago. Not only can these tools now read trillions of base pairs of human DNA every day, but they can be readily repurposed to test for the presence of coronavirus at mass scale, using instruments that already exist across the country. Some methods, such as SHERLOCK and DETECTR, harness CRISPR DNA and RNA recognition tools to enable rapid, distributed testing in doctors offices and at other sites. Other efforts are removing critical bottlenecks, such as sample purification, to make the existing approaches more scalable.

There are additional possibilities, and the US needs to place bets on several of them at the same time. Some of those bets might fail, but the severity of the moment requires that we try. Chances are, we will need more than one of them.

As important as the diagnostic technology itself is the need to fuel innovation at all stages of the testing process, including sample collection, regulation, logistics, manufacturing, distribution, scale-up, data infrastructure, and billing. These are solvable problems. The solutions may sometimes differ from current clinical testing conventions, but these are not conventional times.

Maybe cotton swabs or saliva can be used for collection rather than traditional nasopharyngeal swabs, which are in critically short supply. Maybe mass screening tests dont have to have the tested persons name and date on every collection tube but could instead include a bar code that you snap a picture of with your phone. Maybe these tests can be self-administered at home or work rather than conducted by trained professionals in clinical settings. Maybe samples from low-risk, asymptomatic people can be pooled together for initial testing and further screened only in the event of a positive result. This would allow many more samples to be analyzed at once.

State or federal regulatory agencies could make these adjustments to conventional practices more easily if they were willing to treat mass screening for bringing people back to work differently from the testing used in clinical settings. In addition, mass screening efforts will require unconventional partnerships with private companies, nonprofits, universities, and government agencies to support the logistics, collection, manufacturing, scale-up, and data infrastructure to make such a system possible. All this can be done, and some of it is already starting to be donebut we must not lose hope.

The United States capabilities in the life sciences and information technology are unmatched in the world. The time is now to rapidly build a massively scaled screening program that will save lives while allowing us to reopen our economy and keep it open. This can be done, but it will require urgency and determination to make multiple, simultaneous bets on infrastructure, regulation, and technology, as well as collaboration to put it all together.

We have united before to face far greater challenges as a nation, and we can do so again.

Sri Kosuri is cofounder and CEO of Octant and an associate professor in the Department of Chemistry and Biochemistry at UCLA. Feng Zhang is the James and Patricia Poitras Professor of Neuroscience at MITs McGovern Institute, a core member of the Broad Institute, a Howard Hughes Medical Institute Investigator, and cofounder of Sherlock Biosciences. Jason Kelly is cofounder and CEO of Ginkgo Bioworks. Jay Shendure is a Howard Hughes Medical Institute Investigator at the University of Washington School of Medicine and scientific director of the Brotman Baty Institute.

Originally posted here:
The US already has the technology to test millions of people a day - MIT Technology Review

This Biotechnology ETF Could Be the Place to Be in a Recession – ETF Trends

The ARK Genomic Revolution Multi-Sector Fund (CBOE: ARKG) is already trouncing basic biotechnology and healthcare ETFs and historical data suggest that even if a recession lingers, ARKG could prove durable.

SVB Leerink analyst Geoffrey Porges recently pointed out in a note to clients that biotechnology and pharmaceutical benchmarks topped the broader market during the 2001 recession, the global financial crisis, and during the current economic malaise.

On average, the biotechnology Indexes declined -1% during the three economic downturns, compared with the pharmaceutical indexs -10% and the S&P 500 indexs -20%, reports Josh Nathan-Kazis for Barrons.

ARKG is a credible long-term investment. Over the past three years, the fund is up nearly 105% or more than triple the returns of the Nasdaq Biotechnology Index over the same span. During that period, the ARK fund more than doubled the aforementioned S&P 500 Health Care Index

Some predictable catalysts explain ARKGs potential durability in a recession.

People need their medicine, even in a recession. Porges cited published papers showing that pharmaceutical sales volume stayed steady in the U.S. during the 2008-09 recession, according to Barrons.

The ARK Investment teams process tries to focus on innovation and takes advantage of market inefficiencies. For example, the market easily can be distracted by short-term price movements, losing focus on the long-term effect of disruptive technologies.

Genomic sequencing is changing the way biological information is collected, processed, and applied. ARKG is focused on the disruptive innovations that are increasing precision, restructuring health care, agriculture, pharmaceuticals, and enhancing the quality of life, according to ARK Invest.

The ARK Investment teams process tries to focus on innovation and takes advantage of market inefficiencies. For example, the market easily can be distracted by short-term price movements, losing focus on the long-term effect of disruptive technologies.

Related:Big Growth Awaits a Golden Genomics ETF

Our analysis of historical recessions suggested that the biotech and pharma indices (and stocks) significantly outperformed the broad market (S&P 500), despite the greater P/E multiple compressions in the healthcare indices, writes SVBs Porges.

ARKG also offers some of the best CRISPR exposure of any ETF on the market. CRISPR-based innovations to accelerate given the technologys ease of use, cost-efficacy, a growing body of research surrounding its safety and AI-powered CRISPR nuclease selection tools. CRISPR could also be utilized to address some of the most prominent healthcare problems, which opens up a significant investment opportunity in monogenic diseases.

For more on disruptive technologies, visit our Disruptive Technology Channel.

The opinions and forecasts expressed herein are solely those of Tom Lydon, and may not actually come to pass. Information on this site should not be used or construed as an offer to sell, a solicitation of an offer to buy, or a recommendation for any product.

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This Biotechnology ETF Could Be the Place to Be in a Recession - ETF Trends

Global CRISPR Technology Market Professional Survey 2020 by Manufacturers, Regions, Types and Applications, Forecast to 2024 – Latest Herald

The Global CRISPR Technology Market report vastly covers profiles of the companies who have made it big in this particular field along with their sales data and other data. It also suggests the business models, innovations, growth and every information about the big manufacturers that will be present the future market estimates. The Global CRISPR Technology Market report offers a holistic view of the industry along with the several factors which are driving the Global CRISPR Technology Market. It also shows the possible restraining factors which may hinder the growth of the Global CRISPR Technology Market. The Global CRISPR Technology Market study offers a complete analysis of the market size, segmentation, and market share.

This study covers following key players:

Thermo Fisher ScientificMerck KGaAGenScriptIntegrated DNA Technologies (IDT)Horizon Discovery GroupAgilent TechnologiesCellecta, Inc.GeneCopoeia, Inc.New England BiolabsOrigene Technologies, Inc.Synthego CorporationToolgen, Inc.

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The CRISPR Technology Market study is major compilation of significant information with respect to the competitor details of this market. The data offered in this report is gathered based on the deep market understanding on latest industry news, trends, as well as opportunities. This study offers a separate analysis of the major trends in the existing market, mandates and regulations, micro & macroeconomic indicators is also comprised in this report. By doing so, the study estimated the attractiveness of every major segment during the prediction period.

This analysis report similarly presents the information about present on goings, past results and learnings and in future CRISPR Technology business strategies that have been followed by the key players, company extent, reasons of development and time period, share and estimate analysis having a place with the predicted circumstances and situations that may occur. This analysis report similarly reduces the present, past and in future CRISPR Technology business strategies.

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Market segment by Type, the product can be split into

EnzymesKitsgRNALibrariesDesign Tools

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BiomedicalAgricultural

The CRISPR Technology Market study is major compilation of significant information with respect to the competitor details of this market. The Global CRISPR Technology Market report contains market volume with an accurate estimation offered in the report. Likewise, the information is also inclusive of the several regions where the Global CRISPR Technology Market has successfully gained the position. The Global CRISPR Technology Market report focuses on the major economies, major continents and countries.

The research includes historic data and forecasts which makes the reports a precious supply for the people who are planning to enter into the Global CRISPR Technology Market, executives who look after promotions, consultants of various fields, sales managers, product managers, and many other people considering for significant industry data, willingly accessible documents with deep information on statistics and data presentations through pie charts and graphs which are easy to learn and conclude.

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Global CRISPR Technology Market Professional Survey 2020 by Manufacturers, Regions, Types and Applications, Forecast to 2024 - Latest Herald

Research Roundup: New Protein Linked to Alzheimer’s Identified and More – BioSpace

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

New Protein IDed that May Cause Alzheimers

Scientists at the University of Tokyo tested 19,151 individual genes looking for their effect on amyloid beta levels. Amyloid beta is one of the proteins that accumulates in the brains of Alzheimers patients and is generally viewed as one of the primary drivers of the disease. They identified a new protein using CRISPR/Cas9 gene editing, called calcium and integrin-binding protein 1 (CIB1). They found that cells without functional CIB1 genes generate abnormally high levels of amyloid beta protein. The research was published in FASEB Journal.

We believe this is the first time anyone has used this CRISPR/Cas9 genetic screening technique to look for changes in amyloid beta production, said Yukiko Hori, co-first author and lecturer at the University of Tokyo.

In normal, healthy cells, CIB1 is not directly involved with processing amyloid beta, but it stays attached to another protein, gamma secretase inside cells and at the cell membrane. In cells that dont have CIB1, gamma secretase stays inside the cell longer and doesnt leave the membrane. Amyloid beta undergoes multiple steps before reaching its final form. Normally, gamma secretase processes amyloid beta precursors to help produce the final amyloid beta protein. This happens inside the cell, then gamma secretase moves to the cells outer surface membrane.

Patients diagnosed with early-stage Alzheimers disease have lower levels of CIB1 in their brains, while people with late-stage Alzheimers have higher-than-healthy levels of CIB1.

We cannot say for certain why CIB1 is increased in late-stage Alzheimers disease, said Taisuke Tomita, who runs the research lab where the study was conducted. What is important is that in both the early and late stages of Alzheimers disease, something is abnormal about the regulation of CIB1.

Possible Approach to Improving Gene Therapy

Investigators at the University of Groningen have developed a technique that may improve gene therapies. They use DNA/lipid complexes (lipoplexes). Because the viruses used traditionally in gene therapies can cause an immune response and the cells endosomes tend to degrade DNA or other particles, the lipoplex provides protection. They can fuse with the endosome membrane, which prevents degradation.

Gene Promoters that Can Be Used to Treat Neurological Diseases

Researchers at Princeton Neuroscience Institute have developed new gene promoters that act like switches to turn on gene expression. They can be used in gene therapy, with a particular interest in neurological diseases such as Parkinsons and Alzheimers. Viruses are used to carry genes into cells during gene therapy, typically adeno-associated viruses. The Princeton team used promoters found in herpes viruses, which take up less space than existing promoters and allow the transport of larger genes or multiple genes. They are also long-lasting.

Biosensor to Detect SARS-CoV-2 in the Air

Researchers at Switzerland-based Empa, ETH Zurich and Zurich University Hospital have developed a sensor that has the potential to identify SARS-CoV-2, the novel coronavirus that causes COVID-19, in the air. The work is led by Jing Wang at Empa, who usually works on measuring and analyzing airborne pollutants. The sensor has reliably shown it can identify the first SARS-CoV virus that was responsible for the SARS pandemic in 2003. It has numerous similarities to SARS-CoV-2. Tests showed that the sensor can clearly distinguish between the very similar RNA sequences of the two viruses, Jing Wang said. And the results appear in minutes.

Possible Gene Therapy for Glaucoma

Glaucoma is a common condition of the eye involved fluid buildup in the front part of the eye. It affects more than 64 million people globally and is the leading cause of irreversible blindness. Current treatments include eye drops, laser or surgery. Researchers at the University of Bristol demonstrated that a single injection of a gene therapy using CRISPR and a gene called Aquaporin 1 targeting the ciliary body, where fluid is produced within the eye, led to reduced eye pressure.

More Evidence Parkinsons is an Autoimmune Disease

A study co-led by investigators at the La Jolla Institute for Allergy and Immunology (LJI) adds to the theory that Parkinsons disease is at least partly an autoimmune disease. The research was published in Nature Communications. Science has known for some time that the clumps of a damaged protein known as alpha-synuclein build up in the dopamine-producing brain cells of Parkinsons disease patients. The clumps lead to death of the cells and cause motor symptoms and cognitive decline.

Once these cells are gone, theyre gone, said Cecilia Lindestam Arlehamn, first author of the study and LJI research assistant professor. So if you are able to diagnose the disease as early as possible, it could make a huge difference.

A 2017 study showed that alpha-synuclein attracted certain type of T-cells, causing them to mistakenly attack brain cells, which potentially contributed to the progression of Parkinsons. The new findings found that the T-cells that react to alpha-synuclein are the most abundant when patients are first diagnosed with the disease. They tend to disappear later in the disease and by 10 years after diagnosis, few patients still have them.

This tells us that detection of T-cell responses could help in the diagnosis of people at risk or in early stages of disease development, when many of the symptoms have not been detected yet, said LJI professor Alessandro Sette, who co-led the research with David Sulzer of the Columbia University Medical Center. Importantly, we could dream of a scenario where early interference with T-cell responses could prevent the disease from manifesting itself or progressing.

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Research Roundup: New Protein Linked to Alzheimer's Identified and More - BioSpace

Crispr And Crispr Associated Genes Market Overview, Top Companies, Region, Application and Global Forecast by 2026 – Latest Herald

The world is not only fighting a health pandemic but also an economic one, as the Novel Coronavirus (COVID 19) casts its long shadow over economies around the globe. The complete lockdown situation in several countries, has directly or indirectly impacted many industries causing a shift in activities like supply chain operations, vendor operations, product commercialization, etc. In the latest report on Crispr And Crispr Associated Genes Market, published by Market Research Intellect, numerous aspects of the current market scenario have been taken into consideration and a concise analysis has been put together to bring you with a study that has Pre- and Post-COVID market analysis. Our analysts are watching closely, the growth and decline in each sector due to COVID 19, to offer you with quality services that you need for your businesses. The report encompasses comprehensive information pertaining to the driving factors, detailed competitive analysis about the key market entities and relevant insights regarding the lucrative opportunities that lie in front of the industry players to mitigate risks in such circumstances.

It offers detailed research and analysis of key aspects of the global Crispr And Crispr Associated Genes market. The market analysts authoring this report has provided detailed information on growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Crispr And Crispr Associated Genes market. Market participants can use the market analysis to plan effective growth strategies and prepare for future challenges in advance. Each trend in the global Crispr And Crispr Associated Genes market is carefully analyzed and investigated by market analysts.

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Global Crispr And Crispr Associated Genes Market Segmentation

This market was divided into types, applications and regions. The growth of each segment provides an accurate calculation and forecast of sales by type and application in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting niche markets. Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, the Asia-Pacific region, the Middle East, and Africa and Latin America. Research analysts understand the competitive forces and provide competitive analysis for each competitor separately.

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Crispr And Crispr Associated Genes Market Region Coverage (Regional Production, Demand & Forecast by Countries etc.):

North America (U.S., Canada, Mexico)

Europe (Germany, U.K., France, Italy, Russia, Spain etc.)

Asia-Pacific (China, India, Japan, Southeast Asia etc.)

South America (Brazil, Argentina etc.)

Middle East & Africa (Saudi Arabia, South Africa etc.)

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Executive Summary: In this section, the report focuses on analysis of macroscopic indicators, market issues, drivers, and trends, competitive landscape, CAGR of the global Crispr And Crispr Associated Genes market, and global production. Under the global production chapter, the authors of the report have included market pricing and trends, global capacity, global production, and global revenue forecasts.

Crispr And Crispr Associated Genes Market Size by Manufacturer: Here, the report concentrates on revenue and production shares of manufacturers for all the years of the forecast period. It also focuses on price by manufacturer and expansion plans and mergers and acquisitions of companies.

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Crispr And Crispr Associated Genes Market Overview, Top Companies, Region, Application and Global Forecast by 2026 - Latest Herald

Broad Reply No. 2 to CVC’s Opposition No. 2 to Broad’s Motion No 2 to Substitute the Count – JD Supra

On March 23rd Senior Party The Broad Institute, Harvard University, and the Massachusetts Institute of Technology (collectively, "Broad") filed its Reply to Junior Party the University of California/Berkeley, the University of Vienna, and Emmanuelle Charpentier (collectively, "CVC") Motion No. 2 in Opposition to Broad's Substantive Motion No. 2 to Substitute the Count.

Broad's proposed Count 2 is:

A method, in a eukaryotic cell, of cleaving or editing a target DNA molecule or modulating transcription of at least one gene encoded by the target DNA molecule, the method comprising:contacting, in a eukaryotic cell, a target DNA molecule having a target sequence with an engineered and/or non-naturally-occurring Type II Clustered Regularly lnterspaced Short Palindromic Repeats (CRISPR)-CRISPR associated Cas) (CRISPR-Cas) system comprising: a) a Cas9 protein, and b) RNA comprising i) a targeter-RNA that is capable of hybridizing with the target sequence of the DNA molecule or a first RNA comprising (A) a first sequence capable of hybridizing with the target sequence of the DNA molecule and (B) a second sequence; and ii) an activator-RNA that is capable of hybridizing to the targeter-RNA to form an RNA duplex in the eukaryotic cell or a second RNA comprising a tracr sequence that is capable of hybridizing to the second sequence to form an RNA duplex in the eukaryotic cell,wherein, in the eukaryotic cell, the targeter-RNA or the first sequence directs the Cas9 protein to the target sequence and the DNA molecule is cleaved or edited or at least one product of the DNA molecule is altered.

The distinction Broad made was between embodiments of CRISPR methods that are limited to "single-molecule guide RNA" (aka "fused" or "covalently linked" species), versus embodiments that encompass single-molecule and "dual molecule" species (wherein in the latter versions, the "targeter-RNA" and "activator-RNA" as recited in the proposed Count are not covalently linked). Broad argued that its Proposed Count 2 should be adopted by the Board because it "properly describes the full scope of the interfering subject matter between the parties because both parties have involved claims that are generic, non-limited RNA claims." The brief also argued that Proposed Count 2 "sets the correct scope of admissible proofs [i.e., their own] for the breakthrough invention described by the generic claims at issue in these proceedingsthe successful adaption of CRISPR-Cas9 systems for use in eukaryotic environments," which Broad contended current Court 1 (in either alternative) does not.

Broad's argument in support of its motion was that Count 1 is too narrow for encompassing just a subset of the parties' involved claims. In particular, the brief asserted that most of Broad's involved clams encompass "non-limited" RNA systems and methods. Similarly, the brief argued that CVC itself has many claims directed to non-limited RNA systems and methods and has entire applications that do not recite claims to non-limited RNA systems and methods. Broad asserted that Count 1 does not permit Broad to rely on its earliest and best proofs of invention, which the brief stated is "plainly unfair." This unfairness would preclude Broad from establishing what the brief termed "the fundamental breakthrough - the invention of use of CRISPR in eukaryotic cells" (emphasis in brief). Failing to substitute the Count would instead improperly focus the priority question on who invented the single molecule modification. Colorfully, the brief declared that "[a]llowing the interference to proceed with Count 1 would permit the (single molecule RNA) tail to wag the (breakthrough use of CRISPR in eukaryotic cells) dog."

CVC in its Opposition argued that Proposed Count 2 "goes far beyond converting Count 1 into a generic-guide count." Instead, according to CVC, "it transforms Count 1 into a method so broad that it no longer requires formation of the DNA-targeting complex that includes crRNA, tracrRNA, and Cas9." In addition, according to CVC, Proposed Count 2 does not require that the CRISPR-Cas9 complex even have an effect on the target DNA; rather, it recites that "'a product of the DNA' is altered in some unspecified way" (emphasis in brief), which could include (according to CVC) "alterations to RNA or protein caused by processes that are unrelated to the activity of CRISPR-Cas9" including contamination. And the changes the Broad has effected in Proposed Count 2 "have nothing to do with whether the RNA limitation is single-molecule or generic, Broad's only purported reason for needing a new count" according to CVC.

CVC further argued that the Broad's motion is contrary to the provisions of precedential Board decision, Louis v. Okada, 59 U.S.P.Q.2d 1073 (B.P.A.I. 2001). Under Louis, a party must satisfy a three-prong test: "'(1) should make a proffer of the party's best proofs, (2) show that such best proofs indeed lie outside of the scope of the current count, and (3) further show that the proposed new count is not excessively broad with respect to what the party needs for its best proofs.'" CVC's position (explicated in the brief) is that the Broad failed to provide what Louis required for the "significant alterations" made to Count 1 resulting in Count 2.

The brief summarizes these unnecessary changes as:

"first, Broad has inexplicably eliminated structural and functional limitations that specify the formation of the three-component DNA-targeting complex that includes crRNA, tracrRNA, and Cas9."

"Second, Broad has inexplicably eliminated the requirement that this complex have activity with effects at the DNA level (e.g., cleaving or editing or modulating transcription of DNA). Rather, Proposed Count 2 encompasses merely altering a "productof the DNA molecule" in unspecified ways. Problematically, this breadth includes alterations to downstream products of DNA, such as RNA and protein, that have nothing to do with the activity of the CRISPR-Cas9 system."

"Third, Broad has inexplicably converted Count 1 from a 'cell' or 'system' to a 'method.'"

"Fourth, Broad has inexplicably eliminated the alternative language in CVC's part of Count 1 reciting 'ora nucleic acid comprising a nucleotide sequence comprising . . . .'"

CVC further asserts that the Broad has not shown that Proposed Count 2 is patentable over the prior art.

In its Reply, Broad asserts that CVC did not dispute that the "major advance" at issue is which party invented successful CRISPR in eukaryotic cells, and that this "breakthrough" was not limited to single RNA embodiments of the technology. The brief asserts that current Count 1 "precludes reliance on dual-molecule proofs" (unfairly to Broad) but at the same time this Count "puts at risk all of Broad's claims," which might be considered paradoxical until it is realized that Broad submitted other Motions asking that many if not most of Broad's claims would not correspond to Proposed Count 2.

The brief characterizes CVC's arguments as "nitpick[ing]" and alleges that in CVC's interpretation CRISPR as recited in Count 1 is "so broad it no longer requires a targeting complex 12 that includes crRNA, tracrRNA, and Cas9" (an interpretation that CVC's expert allegedly does not share, which would be curious at least). But even though Broad characterizes these nitpicks as "immaterial" it states that "addressing them would require only small adjustments that could easily be adopted sua sponte by the PTAB." With regard to CVC's purported attempt to limit the scope of the interference to single-molecule embodiments, Broad also asserts that CVC's argued that Broad's 2011 experiments were limited to such embodiments, again arguing that CVC's expert testified to the contrary and characterizing CVC's assertions as being "only attorney argument." The basis for CVC's incorrect arguments in this regard Broad asserts to be an incorrect interpretation of the term "guide RNA" as being limited to single-molecule RNA species.

Broad's synopsis of its reasons for its Motion No. 2 should be granted is:

Broad requests the PTAB to adopt Proposed Count 2 to ensure that, should this interference go forward, claims directed to the broad invention of use of CRISPR-Cas9 in eukaryotic cells, as at issue here, are awarded to the party that first invented use of CRISPR-Cas9 in eukaryotic cells. CVC seeks an interference where claims to use of CRISPR-Cas9 in eukaryotic cells (regardless of type of RNA used) are awarded not to the first inventor of that subject matter, but rather to the party that first created one specific embodiment for which CVC believes it has the best proofs (a single-molecule RNA embodiment). Failing to substitute a generic count for Count 1 would be unjust to Broad and antithetical to the purpose of the Interference, to determine "which of the competing parties was the first to invent the duplicative subject matter." Eli Lilly & Co. v. Bd. of 15 Regents of Univ. of Wash., 334 F. 3d 1264, 1267 (Fed. Cir. 2003) [all emphasis in brief].

Turning to specific arguments against particular features of CVC's brief with which Broad takes issue, the brief (as it must) cites these particular arguments chapter and verse (or more accurately, page and line). The first is that all Broad's claims are directed towards single-molecule embodiments, supported according to Broad solely by attorney argument. Broad argues that both parties have involved claim "indisputably directed to generic RNA guides" (i.e., both single- and dual-molecule guide RNA embodiments). Broad asserts that CVC's misinterpretation of "guide RNA" ignores the plain meaning and "misreads the intrinsic evidence," despite (according to Broad) the use of the term in the Jinik 2012 reference (which Broad states was "perhaps the most important CRISPR publication up to that point and widely read by skilled artisans") as referring to the naturally occurring guide RNA. Broad also asserts that CVC misinterpreted disclosure in its involved patent, which disclosure "does not rise to an 'expression of manifest exclusion or restriction, representing a clear disavowal of claim scope,'" citing Thorner v. Sony Computer Entertainment America LLC, 669 F.3d 1362, 1366 (Fed. Cir. 2012).

The brief also broadly characterizes CVC's criticisms of Proposed Count 2 as "baseless" regarding the four "alleged" differences that "have nothing to do with the single-molecule format of the RNA." Broad says in response that its Proposed Count 2 is "materially the same" as current Count 1 with regard to these four aspects, enumerating the its differences with CVC's interpretation for each:

First, that Proposed Count 2 requires contacting a DNA target with all three components of the CRISPR system (Cas9, crRNA, and tracrRNA) (citing "specific language" of Proposed Count 2 in support);

Second, that Proposed Count 2 requires the occurrence of effects at the target DNA ("cleaving or editing or modulating transcription of DNA") (again relying heavily on CVC's expert's testimony purportedly contrary to CVC's arguments);

Third, that the change from "cell" or "system" in Count 1 to "method" is "immaterial":

Fourth, that eliminating language in Count 1 from Proposed Count 2, recited in the alternative, "a nucleic acid comprising a nucleotide sequence" does not narrow the Count.

Broad also argues that CVC's allegation that Proposed Count 2 is broader than the claims in interference is "based on its erroneous interpretation" of the Proposed Count, which is that the Count does not require tracr RNA (which Broad asserts it does).

With regard to Broad's burden in being granted the relief requested by the PTAB, Broad argues that CVC's challenge regarding Broad's "best proofs" corresponding better to Proposed Count 2 than the current Count are "legally and factually incorrect." Broad supports this allegation by returning to its earlier argument that CVC was wrong in asserting that Broad's earliest eukaryotic application of CRISPR technology was performed with single-molecule guide RNA (calling it "meritless"). The brief sets forth a portion of Inventor Zhang's declaration to illustrate the point:

As Broad argues, this diagram shows three components of CRISPR: Cas9, and separate tracr and crRNAs.

The brief also challenges CVC's argument that Broad had not shown its best proofs are outside the scope of Count 1 (as it is required to so to obtain the requested relief) and that CVC is wrong to assert that Broad was obligated to prove its dual-molecule guide RNA experiments before its single-molecule guide RNA experiments.

The brief specifically addresses CVC's citation of Louis v. Okada, 59 U.S.P.Q.2d 1073 (B.P.A.I. 2001), by asserting that Louis explicitly was not adopted as part of the Board Rules despite a proposal to do so and even if CVC was correct Broad's proffer was sufficient under the rules the PTAB actually adopted.

Finally, Broad argues that CVC did not establish that Broad had failed to show Proposed Count 2 to be patentable; that CVC had not even contested that Broad is not entitled to the benefit of the Zhang B1 reference (its earliest provisional application); and that contrary to CVC's argument a single-molecule Count would not be patentably distinct from a non-limited count.

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Broad Reply No. 2 to CVC's Opposition No. 2 to Broad's Motion No 2 to Substitute the Count - JD Supra

Growth May Be Waiting: At Home Group Inc. (HOME) and CRISPR Therapeutics AG (CRSP) – BOV News

SENVEST MANAGEMENT LLC bought a fresh place in At Home Group Inc. (NYSE:HOME). The institutional investor bought 3.8 million shares of the stock in a transaction took place on 3/02/2020. In another most recent transaction, which held on 3/17/2020, CAS INVESTMENT PARTNERS LLC bought approximately 1.6 million shares of At Home Group Inc. In a separate transaction which took place on 3/31/2020, the institutional investor, NISA INVESTMENT ADVISORS LLC bought 11.6 thousand shares of the companys stock.

In the most recent purchasing and selling session, At Home Group Inc. (HOME)s share price decreased by -3.06 percent to ratify at $1.90. A sum of 1441416 shares traded at recent session and its average exchanging volume remained at 2.62M shares. The 52-week price high and low points are important variables to concentrate on when assessing the current and prospective worth of a stock. At Home Group Inc. (HOME) shares are taking a pay cut of -92.34% from the high point of 52 weeks and flying high of 58.33% from the low figure of 52 weeks.

At Home Group Inc. (HOME) shares reached a high of $1.985 and dropped to a low of $1.75 until finishing in the latest session at $1.90. Traders and investors may also choose to study the ATR or Average True Range when concentrating on technical inventory assessment. Currently at 0.43 is the 14-day ATR for At Home Group Inc. (HOME). The highest level of 52-weeks price has $24.81 and $1.20 for 52 weeks lowest level. After the recent changes in the price, the firm captured the enterprise value of $1.95B. The liquidity ratios which the firm has won as a quick ratio of 0.10, a current ratio of 0.80 and a debt-to-equity ratio of 0.78.

Having a look at past record, were going to look at various forwards or backwards shifting developments regarding HOME. The firms shares fell -2.06 percent in the past five business days and shrunk -6.86 percent in the past thirty business days. In the previous quarter, the stock fell -68.39 percent at some point. The output of the stock decreased -80.90 percent within the six-month closing period, while general annual output lost -91.77 percent. The companys performance is now negative at -65.45% from the beginning of the calendar year.

According to WSJ, At Home Group Inc. (HOME) obtained an estimated Hold proposal from the 9 brokerage firms currently keeping a deep eye on the stock performance as compares to its rivals. 0 equity research analysts rated the shares with a selling strategy, 8 gave a hold approach, 0 gave a purchase tip, 0 gave the firm a overweight advice and 1 put the stock under the underweight category. The average price goal of one year between several banks and credit unions that last year discussed the stock is $2.76.

CRISPR Therapeutics AG (CRSP) shares on Mondays trading session, jumped 3.97 percent to see the stock exchange hands at $53.46 per unit. Lets a quick look at companys past reported and future predictions of growth using the EPS Growth. EPS growth is a percentage change in standardized earnings per share over the trailing-twelve-month period to the current year-end. The company posted a value of $0.97 as earning-per-share over the last full year, while a chance, will post -$4.96 for the coming year. The current EPS Growth rate for the company during the year is 134.10% and predicted to reach at -10.00% for the coming year. In-depth, if we analyze for the long-term EPS Growth, the out-come was 54.00% for the past five years.

The last trading period has seen CRISPR Therapeutics AG (CRSP) move -27.76% and 65.51% from the stocks 52-week high and 52-week low prices respectively. The daily trading volume for CRISPR Therapeutics AG (NASDAQ:CRSP) over the last session is 1.08 million shares. CRSP has attracted considerable attention from traders and investors, a scenario that has seen its volume jump 6.1% compared to the previous one.

Investors focus on the profitability proportions of the company that how the company performs at profitability side. Return on equity ratio or ROE is a significant indicator for prospective investors as they would like to see just how effectively a business is using their cash to produce net earnings. As a return on equity, CRISPR Therapeutics AG (NASDAQ:CRSP) produces 11.70%. Because it would be easy and highly flexible, ROI measurement is among the most popular investment ratios. Executives could use it to evaluate the levels of performance on acquisitions of capital equipment whereas investors can determine that how the stock investment is better. The ROI entry for CRSPs scenario is at 4.90%. Another main metric of a profitability ratio is the return on assets ratio or ROA that analyses how effectively a business can handle its assets to generate earnings over a duration of time. CRISPR Therapeutics AG (CRSP) generated 9.60% ROA for the trading twelve-month.

Volatility is just a proportion of the anticipated day by day value extendthe range where an informal investor works. Greater instability implies more noteworthy benefit or misfortune. After an ongoing check, CRISPR Therapeutics AG (CRSP) stock is found to be 5.84% volatile for the week, while 7.59% volatility is recorded for the month. The outstanding shares have been calculated 59.15M. Based on a recent bid, its distance from 20 days simple moving average is 22.91%, and its distance from 50 days simple moving average is 13.27% while it has a distance of 4.89% from the 200 days simple moving average.

The Williams Percent Range or Williams %R is a well-known specialized pointer made by Larry Williams to help recognize overbought and oversold circumstances. CRISPR Therapeutics AG (NASDAQ:CRSP)s Williams Percent Range or Williams %R at the time of writing to be seated at 15.52% for 9-Day. It is also calculated for different time spans. Currently for this organization, Williams %R is stood at 14.52% for 14-Day, 13.69% for 20-Day, 26.55% for 50-Day and to be seated 43.87% for 100-Day. Relative Strength Index, or RSI(14), which is a technical analysis gauge, also used to measure momentum on a scale of zero to 100 for overbought and oversold. In the case of CRISPR Therapeutics AG, the RSI reading has hit 65.53 for 14-Day.

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Growth May Be Waiting: At Home Group Inc. (HOME) and CRISPR Therapeutics AG (CRSP) - BOV News

What comic book super heroes and villains tell us about plant and human gene editing and the coronavirus – Genetic Literacy Project

Humanity is currently facing a huge challenge imposed by the coronavirus. Borders are being shut down, planes grounded, and factories closed. At the same time, scientists and public health professionals are working on tests, treatments, and vaccines to soon provide a medical response. Coping with corona might be one of the largest tests humans have faced in the past decades but it wont be the last virus we need to defeat. It is time to embrace bioscience and allow more research and applications of genetic alteration methods.

For the layman, all this technobabble about mutagenesis and genetic engineering is difficult to comprehend and it took me personally a good amount of reading to start grasping what different methods exist and how these can massively improve our quality of life.

Lets first look at the four most common ways to alter the genes of a plant or animal:

This can be even done in grown humans that are alive, which is a blessing for everyone who suffers from genetic disorders. We are able to repair genes in live organisms. Gene editing is also thousands of times more accurate than just bombarding seeds with radiation. Some applied examples are deactivating the gene responsible for generating gluten in wheat: The result is gluten-free wheat. There are several methods that achieve this. One of the most popular ones these days is the so-called CRISPR Cas-9. These scissors are usually reprogrammed bacteria that transmit the new gene information or deactivate defunct or unwanted genes. Many science fiction novels and movies show a future in which we can deactivate genetic defects and cure humans from terrible diseases. Some examples of stories in which CRISPR-like techniques have been used are movies such as GATTACA, Star Treks Wrath of Khan, or the Expanse series in which gene editing plays a crucial role in growing crops in space.

Synthetic biologists have started usingCRISPR to synthetically create partsof the coronavirus in an attempt to launch a vaccine against this lung disease and be able to mass-produce it very quickly. In combination with computer simulations and artificial intelligence, the best design for such a vaccine is calculated on a computer and then synthetically created. This speeds up vaccine development and cuts it from years to merely months. Regulators and approval bodies have shown that in times of crisis they can also rapidly approve new testing and vaccination procedures which usually require years of back and forth with agencies such as the FDA?

CRISPR also allows the search for specific genes, also genes of a virus. This helped researchersto build fast and simple testing proceduresto test patients for corona.

In the long term, gene editing might allow us to increase the immunity of humans by altering our genes and making us more resistant to viruses and bacteria.

While the coronavirus seems to really test our modern society, we also need to be aware that this wont be the last pathogen that has the potential to kill millions. If we are unlucky, corona might mutate quickly and become harder to fight. The next dangerous virus, fungus, or bacteria is probably around the corner. Hence we need to embrace the latest inventions of biotechnology and not block genetic research and the deployment of its findings.

Right now a lot of red tape and even outright bans are standing between lifesaving innovations such as CRISPR and patients around the world. We need to rethink our hostility towards genetic engineering and embrace it. To be frank: We are in a constant struggle to fight newly occurring diseases and need to be able to deploy state of the art human answers to this.

Fred Roeder is a Health Economist from Germany and has worked in healthcare reform in North America, Europe, and several former Soviet Republics. One of his passions is to analyze how disruptive industries and technologies allow consumers more choice at a lower cost. Follow him on Twitter @FredCyrusRoeder

A version of this article was originally published at Consumer Choice Center and has been republished here with permission. The center can be found on Twitter @ConsumerChoiceC

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What comic book super heroes and villains tell us about plant and human gene editing and the coronavirus - Genetic Literacy Project

New Covid-19 test could give results in under an hour – but it’s yet to be approved – Health24

Researchers say they've developed a low-cost swab test that can diagnose Covid-19 infections in about 45 minutes.

The CRISPR-based test which uses gene-targeting technology and requires no specialised equipment could help relieve testing backlogs in the United States as Covid-19 continues to spread, the scientists said.

The US Food and Drug Administration has not approved the test, but clinical assessments are being conducted in an effort to fast-track approval. The test is described in a paper published on 16 April in the journal Nature Biotechnology.

"The introduction and availability of CRISPR technology will accelerate deployment of the next generation of tests to diagnose Covid-19 infection," co-lead developer Dr Charles Chiu said in a University of California, San Francisco news release. He is a professor of laboratory medicine at the university.

The new test dubbed SARS-CoV-2 DETECTR is among the first to use CRISPR gene-targeting technology to test for the presence of the novel coronavirus.

CRISPR can be modified to target any genetic sequence, so test developers "programmed" it to zero in on two sequences in the genome of SARS-CoV-2, which causes Covid-19.

One sequence is common to all SARS-like coronaviruses, while the other is unique to SARS-CoV-2. Checking for both sequences ensures that the new test can distinguish between SARS-CoV-2 and closely related viruses, Chiu and his team explained.

Like other tests, this one can detect coronavirus in samples from respiratory swabs from patients. It provides results in about 45 minutes, compared with roughly four hours for widely used tests based on polymerase chain reaction (PCR) techniques.

The researchers said that another advantage of the new test is that it can be performed in virtually any lab, using off-the-shelf chemical agents and common equipment. PCR-based tests require specialised equipment, limiting them to well-equipped diagnostic labs.

The new test is also easy to interpret. Much like a store-bought pregnancy test, dark lines appear on test strips to indicate the presence of coronavirus genes.

While the new test is slightly less sensitive than PCR-based tests, researchers said that's unlikely to have much impact in diagnosis because infected patients typically have high viral loads.

As they work to validate the new test for FDA approval, researchers are making tweaks so that it can be used in field testing at locations such as airports, schools and small clinics.

READ MORE | Physical distancing might need to be practised intermittently until 2022 - study

READ MORE | Coronavirus: The chloroquine debate: Two experts weigh in

READ MORE | Could a measles vaccine help in the fight against Covid-19?

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New Covid-19 test could give results in under an hour - but it's yet to be approved - Health24

Improving berries through CRISPR – hortidaily.com

Pairwise will blend CRISPR gene-editing technology with germplasm of existing berries to create new varieties. Plant Sciences, Inc. (PSI), a berry breeder and ag research company in California, will be Pairwises germplasm provider.

As a result of this fruitful partnership, consumers could see new varieties of black raspberries, red raspberries and blackberries in the supermarkets produce aisle within a few years.

Together, Pairwise and PSI aim to improve berries taste and convenience while also increasing their shelf life and off-season availability.

PSI will use its commercial nurseries to initially grow the new crop plantlets. Pairwise and PSI ultimately will license farmers to plant, grow and produce the new berries.

At Pairwise, we want to make healthy eating easier, said the companys CEO Tom Adams, Ph.D. Now, more than ever, people are focused on their food options and looking for ways to make healthy choices at home. Through the collaboration with PSI, we are moving from science partnerships to product partnerships that will bring new berries to market.

Gene editing and berry (trait) pickingWith CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) gene-editing technology, Pairwise will modify the DNA sequences of berry germplasm supplied by PSI. Pairwise will pick (or retain) good traits and dispose of less desirable traits to cultivate new berry variations.

Pairwise has licensing agreements with Massachusetts General Hospital (MGH) and the Broad Institute of MIT and Harvard for the CRISPR gene-editing technology that makes its berry breeding possible. Pairwise has the exclusive license to specific MGH CRISPR technology for developing agricultural applications.

In a previous interview with the North Carolina Biotechnology Center, Adams explained genomics and data science enable the company to breed the best berry. It is not developing genetically modified, or GMO, berries, which could include adding genes from different organisms.

We really have a mission to drive up consumption of fruits and vegetables through improvements of the crops and making them more available to people, said Adams.

For example, black raspberries have a limited growing season and are not widely available to American consumers. They naturally have five times more antioxidants than blueberries. With genetic modification, black raspberries could grow year-round and become much more available.

The Pairwise/PSI collaboration builds on a unique public/private partnership Pairwise and PSI previously established with the U.S. Department of Agriculture and several leading academic institutions to identify diverse, novel types of berries that are not broadly bred for commercial sale today.

Pairwise is one of the Triangle areas fast-growing agriculture and food biotech companies. Founded in 2018, Pairwise credits the North Carolina Biotechnology Center for being a supportive partner during its startup days.

Now the company is garnering national attention. Its even featured in a new documentary from CNBC Digital,How Scientists Create New Fruits & Vegetables, in which Chief Business Officer Haven Baker is featured in some of the interview segments.

For more information:www.ncbiotech.orgpairwise.comwww.plantsciences.com

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Improving berries through CRISPR - hortidaily.com

Next-generation gene-editing technology: Path to a second Green Revolution? – Genetic Literacy Project

One of the major limitations of the first-generation rDNA-based GM methods is the randomness of DNA insertions into plant genomes, just as the earlier mutagenesis methods introduced mutations randomly. The newer methods increase the specificity and precision with which genetic changes can be made. Known under the general rubric of sequence-specific nuclease (SSN) technology or gene/genome-editing, this approach uses proteins or protein-nucleic acid complexes that bind to and cut specific DNA sequences.1 SSNs include transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs), and meganucleases.2

[This is part three of a four-part series on the progress of agricultural biotechnology. Read part one, part two and part four.]

The DNA cuts made by SSNs are repaired by cellular processes that often either change one to several base pairs or introduce deletions and/or insertions (aka indels) at the target site. Another recently added technology capable of editing gene sequences is termed oligonucleotide-directed mutagenesis (ODM) and uses short nucleic acid sequences to target mutations to selected sites.3

The hottest and the coolest

What is rapidly emerging as the most powerful of the SSN technologies is known by the uninformative acronym CRISPR/Cas, which contracts the unwieldy designation clustered regularly interspaced short palindromic repeats (CRISPR)CRISPR-associated protein (Cas9). Its based on a bacterial defense system against invading viruses and promises extraordinary versatility in the kinds of genome changes that it can make.1,4

The CRISPR/Cas editing molecular machine is comprised of an enzyme (Cas9 and other variants) that binds an RNA molecule (called the guide RNA or gRNA) whose sequence guides the complex to the matching genomic sequence, allowing the Cas9 enzyme to introduce a double-strand break within the matching sequence. The CRISPR/Cas system can be used to edit gene sequences, to introduce a gene or genes at a pre-identified site in the genome, and to edit multiple genes simultaneously, none of which could be done with rDNA methods.1,5

Many of the genetic changes created using either SSN or ODM are indistinguishable at the molecular level from those that occur in nature or are produced by mutation breeding. Since both spontaneous mutants and chemical- and radiation-induced mutants have been used in crop improvement without regulation, there is no scientific rationale for regulating mutants produced by the newer methods. In hopes of creating a distinction that will permit exemption of gene-edited crops from regulation, the newer methods are increasingly referred to as new plant breeding techniques (NPBTs or just NBTs).

Quick successes for NBTs?

Prime targets of gene editing are cellular proteins that are involved in pathogenesis.6 Virus reproduction requires the recruitment of cellular proteins for replication, transcription and translation. There can be sufficient redundancy in the requisite protein infrastructure so that partial or complete virus resistance can be achieved by disrupting genes that code for proteins required for viral replication without damaging crop productivity.

For example, work with mutants of the model plant Arabidopsis identified translation initiation factor eIF4E as required for potyvirus translation. CRISPR/Cas-induced point mutations and deletions have recently been reported to enhance viral resistance not only in Arabidopsis, but in cucumber and cassava, as well.7

The many ways that plants and their bacterial and fungal pathogens interact offer opportunities to use gene editing to enhance plant disease resistance and reduce agricultures dependence on chemical control agents.6 The two main strategies are to inactivate genes whose products render the host plant sensitive to pathogen invasion and to enhance the ability of the host plant to resist invasion by providing functional resistance factors they lack.

An example of the former is provided by the mildew resistance resulting from the inactivation of all three homeoalleles of the mildew resistance locus (MLO) of hexaploid wheat.8 The efficiency of targeting both multiple alleles and multiple loci has taken a further jump with the development of multiplexed gene editing using vectors carrying several gRNA sequences capable of being processed by cellular enzymes to release all of them. This allows the gRNAs to edit multiple genes simultaneously.9

The second approach is to capitalize on the formidable arsenal of resistance genes residing in plant genomes.10 Fungal resistance genes have long been a major target of breeders efforts and have proved frustratingly short-lived, as pathogens rapidly evolve to evade recognition.11 While desirable resistance genes missing from domesticated crops still reside in wild relatives, extracting them by conventional breeding methods can be time-consuming or impossible.

European academic researchers created transgenic potatoes resistant to the late blight (Phytophthora infestans) that caused the Irish potato famine by inserting resistance (R) genes cloned from wild potato species into commercial potato varieties.12 A blight-resistant variety, called the InnateTM Generation 2 potato, is being commercialized by J.R. Simplot company in the U.S. and Canada and is already being marketed in the U.S. as the White Russet TM Idaho potato.13 Transgenic disease-resistance traits have been introduced in other crops, but have yet to be commercialized.14

Plant genomes contain hundreds to thousands of potential R genes, but it is not yet possible to determine whether a given one will confer resistance to a particular pathogen. Methods are currently being developed to accelerate the identification and cloning of active ones.14 Once identified, CRISPR/Cas can be used to introduce cassettes carrying multiple R genes, making it possible to create more durable resistance than can be achieved by introducing a single R gene through conventional breeding14. Finally, direct editing of resident inactive R genes using a ribonucleoprotein (RNP) strategy that avoids creating a transgenic plant may prove useful, although no such products appear to be in the pipeline to commercialization at present.15,16

Multiplexed editing has proved particularly useful for editing genes in polyploid species. For example, Cas9/sgRNA-mediated knockouts of the six fatty acid desaturase 2 (FAD2) genes of allohexaploid Camelina sativa was reported to markedly improve the fatty acid composition of Camelina oil.17 Using a different approach, Yield10 Biosciences is moving toward commercialization of a high-oil Camelina developed by editing a negative regulator of acetyl-CoA carboxylase.18

As of this writing, the only gene-edited product that has been commercialized is a soybean oil with no trans-fat, trademarked CalynoTM, developed by Calyxt.19 Gene-edited crops that have been approved but not commercialized or are still in the regulatory pipeline include miniature tomatoes, high-fiber wheat, high-yield tomatoes, improved quality alfalfa, non-browning potatoes and mushrooms, as well as high starch-content and drought-resistant corn, most being developed by small biotech companies.19

Getting beyond the low-hanging fruit

It is becoming increasingly clear that yield increases in our major crops by traditional breeding approaches are not keeping pace with demand.20 The gap is likely to widen as climate warming moves global temperatures farther from those prevailing when our crops were domesticated.

Overexpression of stress-related transcription factors has been reported to increase yields under water-stress conditions, but such increases are generally not maintained under optimal conditions.21 Monsantos drought-tolerant (Genuity DroughtGardTM) corn hybrids are based on the introduction of bacterial chaperone genes.22 Fortunately, research into drought stress tolerance in wheat and other grains continues apace, although no drought-tolerant varieties have yet reached farmers.23

Real progress on crop yield is slow. What stands in the way is that we have so limited an understanding of how plants work at the molecular level. At every level of analysis, organisms are redundant networks of interconnected proteins that adjust their manifold physical and enzymatic interactions in response to internal signals and external stimuli, then send messages to the information storage facilities (DNA) to regulate their own production and destruction rates.

As well, many genes are present in families of between two and hundreds or thousands of similar members, making it difficult to determine either the function or the contribution of any given member to a complex trait such as stress tolerance or yield. That said, gene family functions are identifiable and some, such as transcription factor genes, encode proteins that influence multiple other genes, making them among the likeliest candidates for manipulation. Indeed, studies on the genetics of domestication often point to changes in transcription factor genes.24

But while there have been reports that constitutive overexpression of single transcription factor gene can increase grain yield in both wheat and maize, none appear to have been commercialized yet.25 The challenge of developing a yield-improved variety by simply overexpressing transcription factor genes is illustrated by a recent report from Corteva.26 It describes a tour-de-force involving generation and testing of countless transgenic plants to identify a single transcription factor gene, ZMM28, that reproducibly increased yield when incorporated into 48 different hybrids and tested over a 4-year period in 58 locations.26

Getting there by a different route

Might gene-editing facilitate the task of generating and identifying yield-enhancing genetic variation? While the CRISPR/Cas toolkit is growing at dizzying speed, its utility in crop improvement has so far been limited to the simple traits controlled by individual genes, albeit including multiple alleles.1,27

Crop domestication and plant breeding have vastly narrowed genetic diversity because the very process of selecting plants with enhanced traits imposes a bottleneck, assuring that only a fraction of the ancestral populations genetic diversity is represented in a new elite variety. This, in turn, limits what can be done by mutagenizing existing elite varieties, a process that is also burdened with the necessity to eliminate deleterious mutations through back-crossing.

But to widen the genetic base and to modify genes that contribute to quantitative traits, it is still first necessary to identify the genes that contribute to agronomically important traits. Identifying such genes is currently a slow and tedious process of conventional and molecular mapping.28 A recent report describes a method for combining pedigree analysis with targeted CRISPR/Cas-mediated knockouts that promises to markedly accelerate the identification of the individual contributing genes in the chromosomal regions that are associated with quantitative traits, technically known as quantitative trait loci (QTLs).29

Even as the QTL knowledge gap narrows, gRNA multiplexing is extending the power of SSNs to understanding and modifying complex traits in crop plants. For example, using multiplexed gRNAs, Cas nuclease was simultaneously targeted to three genes known to be negative regulators of grain weight in rice.30 The triple mutants were reported to exhibit increases in the neighborhood of 25% in each of the three grain weight traits: length, width and thousand grain weight.

In another study, 8 different genes affecting rice agronomic traits were targeted with a single multiplexed gRNA construct and all showed high mutation efficiencies in the first generation.31 Conversely, it has been reported that editing the same QTLs gives different outcomes in different elite varieties, improving yield in some but not other.32

Mutations affecting the expression of regulatory genes, such as transcription factors genes, account for a substantial fraction of the causative genetic changes during crop domestication.33 Multiplexed gRNAs constructs targeting cis-regulatory elements (CREs) have been used to generate large numbers of allelic variants of genes affecting fruit size in tomato, mimicking some of the mutations accumulated during domestication and breeding of contemporary tomato varieties.34

Knowledge of domestication genes can also be used to accelerate domestication of wild plants that retain traits of value, such as salt tolerance, as reported for tomato.35 This opens the possibility of rapidly domesticating wild species better adapted to the harsher climate conditions of the future.

While the above-described advances have been based on the CRISPR/Cas-mediated deletions, approaches to more precise sequence editing are developing as well. While Cas-generated cuts in the DNA are most commonly repaired by the non-homologous end joining pathway (NHEJ), the less frequent homology-directed repair pathway (HDR) has been shown to edit sequences at useful frequencies using Cas-gRNA ribonucleoprotein complexes.15,36

As well, mutant Cas9 proteins lacking nuclease activity have been fused with base-editing enzymes such as cytidine and adenosine deaminases to direct gene editing without DNA cleavage.37,38 This approach can change single base pairs precisely in both coding and non-coding regions, as well alter mRNA precursor processing sites.38 Finally, the sequence targeting properties of the CRISPR-Cas system can be used to deliver other types of hybrid proteins to target sequences to regulate gene expression and DNA methylation.27

In sum, the many variations on gene editing now developing hold the promise of revolutionizing crop breeding, prompting several colleagues to whimsically title a recent review of CRISPR/Cas-based methodology: Plant breeding at the speed of light.39 And indeed, the new methods make it possible to replace chemicals with biological mechanisms in protecting plants from pests and disease, as well as increase their resilience to stress.

That said, extraordinary progress in increasing grain yields has already been accomplished by what are now considered to be traditional breeding methods and increased fertilizer use. Further improvements continue, but will likely be harder won than the many-fold increases in corn, wheat and rice yields of the last century and its Green Revolution. But there is a persistent disconnect between what can be done to accelerate plant breeding using the new gene-editing toolkit and what is actually being done by both the public and private sectors to get varieties improved by these methods out to farmers.

1Zhang Y et al. (2019). The emerging and uncultivated potential of CRISPR technology in plant science. Nature Plants 5:778-94.

2Podevin N et al. (2013). Site-directed nucleases: a paradigm shift in predictable, knowledge-based plant breeding. Trends Biotechnol 31:375-83.

3Sauer NJ et al. (2016). Oligonucleotidedirected mutagenesis for precision gene editing. Plant Biotechnol J 14:496-502.

4Zhang D et al. (2016). Targeted gene manipulation in plants using the CRISPR/Cas technology. J Genet Genomics 43:251-62.

5Cong L et al. (2013). Multiplex genome engineering using CRISPR/Cas systems. Science 339:819-23.

6Borrelli VM et al. (2018). The enhancement of plant disease resistance using CRISPR/Cas9 technology. Frontiers Plant Sci 9:Article 1245.

7Chandrasekaran J et al. (2016). Development of broad virus resistance in nontransgenic cucumber using CRISPR/Cas9 technology. Molec Plant Pathol 17:1140-53; Pyott DE et al. (2016). Engineering of CRISPR/Cas9mediated potyvirus resistance in transgenefree Arabidopsis plants. Molec Plant Pathol 17:1276-88; Gomez MA et al. (2019). Simultaneous CRISPR/Cas9mediated editing of cassava eIF 4E isoforms nCBP1 and nCBP2 reduces cassava brown streak disease symptom severity and incidence. Plant Biotechnol J 17:421-34.

8Wang Y et al. (2014). Simultaneous editing of three homoeoalleles in hexaploid bread wheat confers heritable resistance to powdery mildew. Nature Biotechnol 32:947.

9Xie K et al. (2015). Boosting CRISPR/Cas9 multiplex editing capability with the endogenous tRNA-processing system. Proc Natl Acad Sci 112:3570-5; Wang W et al. (2018). Transgenerational CRISPR-Cas9 activity facilitates multiplex gene editing in allopolyploid wheat. The CRISPR J 1:65-74.

10Petit-Houdenot Y and Fudal I (2017). Complex interactions between fungal avirulence genes and their corresponding plant resistance genes and consequences for disease resistance management. Frontiers Plant Sci 8:1072.

11Bebber DP and Gurr S (2015). Crop-destroying fungal and oomycete pathogens challenge food security. Fungal Genet Biol 74:62-4; van Esse HP et al. (2020). Genetic modification to improve disease resistance in crops. New Phytol 225:70-86.

12Jones JD et al. (2014). Elevating crop disease resistance with cloned genes. Phil Trans Royal Soc B: Biol Sci 369:20130087; Haesaert G et al. (2015). Transformation of the potato variety Desiree with single or multiple resistance genes increases resistance to late blight under field conditions. Crop Protection 77:163-75.

13Halsall M. Innate outlook. Spudsmart, 24 April 2019 https://spudsmart.com/innate-outlook/

14Dong OX and Ronald PC (2019). Genetic engineering for disease resistance in plants: recent progress and future perspectives. Plant Physiol 180:26-38.

15Svitashev S et al. (2016). Genome editing in maize directed by CRISPRCas9 ribonucleoprotein complexes. Nature Communications 7:1-7.

16Mao Y et al. (2019). Gene editing in plants: progress and challenges. Nat Sci Rev 6:421-37.

17Morineau C et al. (2017). Selective gene dosage by CRISPRCas9 genome editing in hexaploid Camelina sativa. Plant Biotechnol J 15:729-39; Jiang WZ et al. (2017). Significant enhancement of fatty acid composition in seeds of the allohexaploid, Camelina sativa, using CRISPR/Cas9 gene editing. Plant Biotechnol J 15:648-57.

18Yield10 Bioscience (Jan 16, 2020 ). Yield10 Bioscience submits Am I Regulated? letter to USDA-APHIS BRS for CRISPR genome-edited C3007 in Camelina to pave the way for U.S. field tests. https://www.globenewswire.com/news-release/2020/01/16/1971418/0/en/Yield10-Bioscience-Submits-Am-I-Regulated-Letter-to-USDA-APHIS-BRS-for-CRISPR-Genome-Edited-C3007-in-Camelina-to-Pave-the-Way-for-U-S-Field-Tests.html

19Genetic Literacy Project (2020). Global Gene Editing Regulation Tracker. https://crispr-gene-editing-regs-tracker.geneticliteracyproject.org/united-states-crops-food/

20Ray DK et al. (2013). Yield trends are insufficient to double global crop production by 2050. PloS One 8:e66428.

21Rice EA et al. (2014). Expression of a truncated ATHB17 protein in maize increases ear weight at silking. PLoS One 9:e94238; Araus JL et al. (2019). Transgenic solutions to increase yield and stability in wheat: shining hope or flash in the pan? J Experimental Bot 70:1419-24.

22Castiglioni P et al. (2008). Bacterial RNA chaperones confer abiotic stress tolerance in plants and improved grain yield in maize under water-limited conditions. Plant Physiol 147:446-55.

23Mwadzingeni L et al. (2016). Breeding wheat for drought tolerance: Progress and technologies. J Integrative Agricult 15:935-43; Sallam A et al. (2019). Drought stress tolerance in wheat and barley: Advances in physiology, breeding and genetics research. Internat J Mol Sci 20:3137.

24Swinnen G et al. (2016). Lessons from domestication: targeting cis-regulatory elements for crop improvement. Trends Plant Sci 21:506-15.

25Nelson DE et al. (2007). Plant nuclear factor Y (NF-Y) B subunits confer drought tolerance and lead to improved corn yields on water-limited acres. Proc Natl Acad Sci 104:16450-5; Qu B et al. (2015). A wheat CCAAT box-binding transcription factor increases the grain yield of wheat with less fertilizer input. Plant Physiol 167:411-23; Yadav D et al. (2015). Constitutive overexpression of the TaNF-YB4 gene in transgenic wheat significantly improves grain yield. J Experiment Bot 66:6635-50.

26Wu J et al. (2019). Overexpression of zmm28 increases maize grain yield in the field. Proc Natl Acad Sci 116:23850-8.

27Chen K et al. (2019). CRISPR/Cas genome editing and precision plant breeding in agriculture. Annu Rev Plant Biol 70:667-97.

28Cavanagh C et al. (2008). From mutations to MAGIC: resources for gene discovery, validation and delivery in crop plants. Curr Opin Plant Biol 11:215-21.

29Huang J et al. (2018). Identifying a large number of high-yield genes in rice by pedigree analysis, whole-genome sequencing, and CRISPR-Cas9 gene knockout. Proc Natl Acad Sci 115:E7559-E67.

30Xu R et al. (2016). Rapid improvement of grain weight via highly efficient CRISPR/Cas9-mediated multiplex genome editing in rice. J Genet Genom 43:529.

31Shen L et al. (2017). Rapid generation of genetic diversity by multiplex CRISPR/Cas9 genome editing in rice. China Sci Life Sci 60:506-15.

32Shen L et al. (2018). QTL editing confers opposing yield performance in different rice varieties. J Integrative Plant Biol 60:89-93; Zhou J et al. (2019). Multiplex QTL editing of grain-related genes improves yield in elite rice varieties. Plant Cell Rep 38:475-85.

33Meyer RS and Purugganan MD (2013). Evolution of crop species: genetics of domestication and diversification. Nature Rev Genet 14:840-52.

34Rodrguez-Leal D et al. (2017). Engineering quantitative trait variation for crop improvement by genome editing. Cell 171:470-80. e8.

35Li T et al. (2018). Domestication of wild tomato is accelerated by genome editing. Nature Biotechnol 36:1160-3; Zsgn A et al. (2018). De novo domestication of wild tomato using genome editing. Nature Biotechnol 36:1211-6.

36Puchta H et al. (1996). Two different but related mechanisms are used in plants for the repair of genomic double-strand breaks by homologous recombination. Proc Natl Acad Sci 93:5055-60; Zhang Y et al. (2016). Efficient and transgene-free genome editing in wheat through transient expression of CRISPR/Cas9 DNA or RNA. Nature Communications 7:1-8.

37Komor AC et al. (2016). Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 533:420-4; Hua K et al. (2019). Expanding the base editing scope in rice by using Cas9 variants. Plant Biotechnol J 17:499-504.

38Kang B-C et al. (2018). Precision genome engineering through adenine base editing in plants. Nature Plants 4:427-31.

39Wolter F et al. (2019). Plant breeding at the speed of light: the power of CRISPR/Cas to generate directed genetic diversity at multiple sites. BMC Plant Biol 19:176.

Nina V. Fedoroff is an Emeritus Evan Pugh Professor at Penn State University

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CRISPR-Based ‘Discovery Engine’ for New Cell Therapies to Advance Cancer Treatments – UCSF News Services

Despite centuries-long efforts to develop cures for cancer, various forms of the disease will kill about 630,000 people in the U.S. in 2020. But hopes are rising for cell therapies sometimes called living medicines that can boost and adapt the natural cancer-fighting potential of the immune system in ways that conventional cancer treatments cannot match.

UC San Francisco researchers now have reported a new method to design and test cell therapies, one they expect will speed the development of new life-saving treatments not only for cancer, but for other diseases, too.

Cell therapy for cancer is a type of immunotherapy. Immune-system cells known as T cells are isolated from a patients blood and genetically modified in the lab, inserting or removing genes so the cells will better recognize and destroy tumors. These modified T-cells are grown in culture until they number in the hundreds of millions, and are then infused back into the patient through an IV drip.

One form of cell therapy, known as CAR-T-cell therapy, is already approved for certain blood cancers, but so far cell therapies have not been effective against the solid tumors that affect the breast, colon, brain, lung, and other tissues.

In 2018, a UCSF team led by immunologist Alex Marson, MD, PhD, along with Theo Roth, an MD/PhD student in the UCSF Medical Scientist Training Program, developed a breakthroughtechnique in which they used pulses of electricity (a method called electroporation) to enable CRISPR gene-targeting technology to quickly and efficiently reprogram T-cells with new functions.

Now, in a study published April 16, 2020 in Cell, the team hasadvanced this technique to power a high-throughput platform to evaluate the specificity and potency of many different potential cell therapies simultaneously comparable to the approach already widely used in industry to quickly screen large batches of small molecules to assess whether they would make effective drugs.

The UCSF researchers evaluated a library of 36 different DNA sequences, a selection based on educated guesses about which bits of genetic material, when spliced into T cells, might alter cell function to better fight cancer. They inserted the different sequences into different T cells to compare their best guesses head to head in the lab.

Rather than pursuing one educated guess at a time, we wanted a systematic way to compare different potential therapeutic edits head to head, said Marson, who serves as scientific director of biomedicine at the UCSFUC Berkeley Innovative Genomics Institute (IGI) and is a Chan Zuckerberg Biohub Investigator. The ability to iterate fast and test different candidates against each other is what the field needs to move forward and have a discovery engine for next-generation cell therapies.

Many solid tumors express proteins that shield them from T cell attacks, so the researchers evaluated not only the modified cells ability to find cancer cells, but also their ability to accumulate robustly within tumors. Using a distinctive DNA barcode to track the modified T cells, the researchers raced them against one another in lab dishes and in vivo cancer models. This allowed them to identify specific gene modifications that give T cells the best ability to kill off solid-tumor cells in a lab dish and also more effectively fight a type of human skin cancer grown in mice.

Their new methods also enable researchers to measure specific patterns of gene activation within individual T cells to gain insight into how cellular functions have changed in the modified cells that fare best against tumors.

We now are working to further scale up and optimize this screening platform, said Roth, who with Marson is a co-founder of Arsenal Biosciences, a company funded in part by the Parker Institute for Cancer Immunotherapy that is focused on producing and testing new cell therapies. We believe this cell therapy development platform will make it possible for academics and industry to begin generating all sorts of genetically programed T cells targeted to specific cancers as well as other medical applications.

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CRISPR-Based 'Discovery Engine' for New Cell Therapies to Advance Cancer Treatments - UCSF News Services

Food Tech News: CRISPR Blackberries and a New Nordic FoodTech Fund – The Spoon

Are you baking bread this weekend? (Hot tip: Even if you cant find yeast at the store, theres a simple way to make your own at home.)

In between your dough prooves is a great time to catch up on your latest dose of food tech news. This week weve got stories on fresh varietals of gene-edited berries, a new Nordic FoodTech VC fund, Burger Kings trouble over its plant-based burger ads in the UK, and more.

Pairwise partners to breed new type of berriesAgriculture and biotech company Pairwise forged a partnership with Plant Sciences Inc (PSI) this week to create new types of berries (via WRAL TechWire). Financial terms of the deal were not disclosed. Pairwise uses CRISPR gene editing to develop new varietals of food that are optimized for nutrition, have longer shelf lives or grow more quickly. First up, Pairwise and PSI will focus on black and red raspberries, as well as blackberries. Theyre hoping to have their first round of berries on shelves within the next few years.

Lyft launches delivery program for orgs affected by COVID-19Rideshare and last-mile logistics company Lyft launched a new COVID-19-related initiative this week. Essential Deliveries is a program that partners with businesses and nonprofits to help them deliver staple goods like groceries, prepared meals, and cleaning and medical supplies (h/t Techcrunch) to consumers. Partners can tap into Lyfts platform to set up deliveries or schedule rides. The program will be available in at least 11 cities nationwide and drivers will be alerted about the nature of the goods theyre delivering. All deliveries will be contact-free.

Nordic FoodTech VC launches with 24.55 millionNordic FoodTech VC, a new venture fund targeting early-stage tech companies making the food system more sustainable and nutritious, has launched this week. The fund will begin investing with 24.55 million ($26.7 million USD) in capital. Its the first fund in the Nordic countries and plans to invest in dozens of companies innovating to improve the global food system.

Burger Kings plant-based Whopper ads banned in UKThree ads from Burger King in the UK promoting its Rebel Whopper have now been banned by the UKs Advertising Standards Authority. Burger King launched the Rebel Whopper, which features a plant-based burger from Unilever-owned Vegetarian Butcher, back in January 2020. Since then, complaints came in stating that the ad was misleading consumers by suggesting that it could be eaten by vegetarians, vegans, and people with egg allergies, despite the fact that its cooked on the same grill as meat products and features mayonnaise. The ASA has sided with the complaints, stating that the small print at the bottom of BKs ads stating that the Rebel Whopper is cooked alongside meat products was not sufficiently in informing consumers.

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CRISPR Therapeutics AG’s (NASDAQ:CRSP) Financials Are Too Obscure To Link With Current Share Price Momentum: What’s In Store For the Stock? – Yahoo…

Most readers would already be aware that CRISPR Therapeutics' (NASDAQ:CRSP) stock increased significantly by 30% over the past month. But the company's key financial indicators appear to be differing across the board and that makes us question whether or not the company's current share price momentum can be maintained. Particularly, we will be paying attention to CRISPR Therapeutics' ROE today.

Return on equity or ROE is an important factor to be considered by a shareholder because it tells them how effectively their capital is being reinvested. In simpler terms, it measures the profitability of a company in relation to shareholder's equity.

See our latest analysis for CRISPR Therapeutics

Return on equity can be calculated by using the formula:

Return on Equity = Net Profit (from continuing operations) Shareholders' Equity

So, based on the above formula, the ROE for CRISPR Therapeutics is:

7.1% = US$67m US$939m (Based on the trailing twelve months to December 2019).

The 'return' is the income the business earned over the last year. That means that for every $1 worth of shareholders' equity, the company generated $0.07 in profit.

Thus far, we have learnt that ROE measures how efficiently a company is generating its profits. Based on how much of its profits the company chooses to reinvest or "retain", we are then able to evaluate a company's future ability to generate profits. Generally speaking, other things being equal, firms with a high return on equity and profit retention, have a higher growth rate than firms that dont share these attributes.

On the face of it, CRISPR Therapeutics' ROE is not much to talk about. Next, when compared to the average industry ROE of 19%, the company's ROE leaves us feeling even less enthusiastic. Therefore, it might not be wrong to say that the five year net income decline of 24% seen by CRISPR Therapeutics was probably the result of it having a lower ROE. We reckon that there could also be other factors at play here. For instance, the company has a very high payout ratio, or is faced with competitive pressures.

That being said, we compared CRISPR Therapeutics' performance with the industry and were concerned when we found that while the company has shrunk its earnings, the industry has grown its earnings at a rate of 24% in the same period.

NasdaqGM:CRSP Past Earnings Growth April 18th 2020

Earnings growth is an important metric to consider when valuing a stock. What investors need to determine next is if the expected earnings growth, or the lack of it, is already built into the share price. By doing so, they will have an idea if the stock is headed into clear blue waters or if swampy waters await. One good indicator of expected earnings growth is the P/E ratio which determines the price the market is willing to pay for a stock based on its earnings prospects. So, you may want to check if CRISPR Therapeutics is trading on a high P/E or a low P/E, relative to its industry.

CRISPR Therapeutics doesn't pay any dividend, meaning that the company is keeping all of its profits, which makes us wonder why it is retaining its earnings if it can't use them to grow its business. So there could be some other explanations in that regard. For instance, the company's business may be deteriorating.

Story continues

In total, we're a bit ambivalent about CRISPR Therapeutics' performance. While the company does have a high rate of reinvestment, the low ROE means that all that reinvestment is not reaping any benefit to its investors, and moreover, its having a negative impact on the earnings growth. That being so, the latest industry analyst forecasts show that analysts are forecasting a slight improvement in the company's future earnings growth. This could offer some relief to the company's existing shareholders. Are these analysts expectations based on the broad expectations for the industry, or on the company's fundamentals? Click here to be taken to our analyst's forecasts page for the company.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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CRISPR Therapeutics AG's (NASDAQ:CRSP) Financials Are Too Obscure To Link With Current Share Price Momentum: What's In Store For the Stock? - Yahoo...

Satyajit Rays Feluda will soon detect coronavirus in minutes, thanks to CSIR scientists – ThePrint

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New Delhi: Feluda will now detect if you are infected with Covid-19. Or, to be precise, a new paper-based test strip named after the beloved detective character created by Bengali filmmaker-author Satyajit Ray will soon be able to find the novel coronavirus in just a few minutes.

The Feluda test strip has been invented by a team led by two Bengali-origin scientists Dr Souvik Maiti and Dr Debojyoti Chakraborty at the Council of Scientific & Industrial Researchs Institute of Genomics and Integrative Biology (CSIR-IGIB) in New Delhi.

The simple paper-based test strip could also reduce Covid-19 testing costs the real-time polymerase chain reaction test (RT-PCR) used currently requires machinery worth lakhs of rupees and its price is capped at Rs 4,500 in private labs, but the Feluda test could cost as little as Rs 500. It can be used in a way similar to pregnancy test strips widely available over the counter.

This strip will be similar to a pregnancy test strip, and will not require any specialised skill and machines to perform, as is the case with other PCR-based tests. This strip will just change colour, and can be used in a simple pathological lab. The most important part is it will be 100 per cent accurate, CSIR Director-General Shekhar C. Mande told ThePrint.

Normally, scientists take two to three years to develop this type of kits, but we are among the three or four countries leading the way in developing it, alongside Stanford University and Massachusetts Institute of Technology (MIT) in the US, said Mande, head of Indias largest umbrella organisation for scientific research.

Also read: Harsh Vardhan asks scientists to hurry up: Deliver Covid-19 weapons before the war is over

Dr Chakraborty of IGIB explained how his team had developed the kit.

We were experimenting on sickle cell anaemia for the last two years. When Covid-19 cases rose in China, we started to experiment to see how mutations take place in the coronavirus. For the last two months, we have been working 20 hours a day to develop it, he told ThePrint.

Asked why they named it after Rays fictional detective, Chakraborty said: It will detect the presence of a virus in a just few minutes,like Feluda.

The team is currently testing the sensitivity of the Feluda strip. Now, we are at a stage where we can say it will be a major breakthrough for testing in a short time. Regulatory validation is in process, and we hope we will be ready for technology transfer in few weeks. We are in touch with several manufacturers for the technology transfer, Chakraborty said.

Mande added that the strip has been tested on the samples with CSIR, and is now being tested on samples from elsewhere to find out its sensitivity.

Also read: FDA has approved saliva test to detect Covid-19 that lowers infection risk for health staff

Mande and Chakraborty said their test kit uses CRISPR gene-editing technology to get results, though the difference to the kits being developed at Stanford and MIT is in the proteins used.

CRISPR technology recognises specific genetic sequences and cuts them in short time. The CRISPR reaction is specific, and can be done in 5-10 minutes. It is a powerful technique that worked in detecting the Zika virus too.

(Our strip) uses cutting-edge gene-editing CRISPR-CAS-9 technology to target and identify genomic sequence of the novel coronavirus in suspected individuals. No other laboratory in India is developing test kit using CRISPR technology, Mande said.

Chakraborty added: Unlike Stanford and MIT, which use CAS-12 and CAS-13 proteins to detect the presence of the novel coronavirus, our kit uses CAS-9 protein technology. And unlike the PCR test, there is no need for probes.

Anurag Agrawal, director of CSIR-IGIB, explained the difference between the Feluda paper strip test and others being carried out around the world.

A few other labs have been developing test kits, but they are largely based on PCR technology. The problem with PCR is that it is costly one machine costs Rs 14-15 lakh, and imported probes have to be used, of which there is a shortage. It takes several hours, Agrawal said.

Our paper strip does not require any level 2 or level 3 lab to test, unlike most PCR-based tests. This can be done in any simple pathological lab. We have imported serological rapid test kits, but this paper test technology is different, Agrawal added.

Also read: US begins clinical trial of an artificial antibody for Covid-19 treatment

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Next Generation Of COVID-19 Virus Tests Could Get Faster And Cheaper With CRISPR : Shots – Health News – NPR

Mammoth Biosciences and researchers at the University of California, San Francisco are working on a coronavirus test that could run multiple samples at once, with results in 35-40 minutes. Even better, they say, it doesn't require the sophisticated, expensive equipment used in other tests for the virus. Mammoth Biosciences hide caption

Mammoth Biosciences and researchers at the University of California, San Francisco are working on a coronavirus test that could run multiple samples at once, with results in 35-40 minutes. Even better, they say, it doesn't require the sophisticated, expensive equipment used in other tests for the virus.

Being able to test for coronavirus infections is a critical component to reopening society even a little bit after the initial wave of COVID-19. So there is an urgent need for faster, cheaper tests than the ones available at present.

One approach to the next generation of tests is being developed by the University of California, San Francisco Medical School and Mammoth Biosciences. In a paper released Thursday in the journal Nature Biotechnology, researchers describe a test based on a new technology known as CRISPR.

CRISPR systems have been widely used by researchers to modify the genetic material in living cells. In this case, a system known as CRISPR-Cas12 is used to recognize genetic signatures of the coronavirus that causes COVID-19 and then make cuts in it to release a fluorescent molecule that will show whether the virus is present.

Like the test developed by the Centers for Disease Control and Prevention, this CRISPR-based test can run multiple samples at once. And while the CDC version delivers answers in hours, the test from UCSF and Mammoth Biosciences is faster providing results in 30-45 minutes.

The test is self-contained, so it doesn't require sophisticated, expensive equipment that is used in other tests for the virus.

"I can run it now myself at home," explains Dr. Charles Chiu, professor of laboratory medicine at UCSF and co-lead developer of the new test although he notes it does require some expertise to conduct it. He says he and his colleagues hope to submit the current version of their test next week for FDA approval. But it probably won't be the final iteration.

"What we really want to develop is something like a handheld, pocket-sized device using disposable cartridges," says Chiu something that could even be use by nonexperts as a home-based test. Chiu is confident such tests could be manufactured at a scale that would be widely available.

Other labs, including two at the Broad Institute in Cambridge, Mass., are also working on CRISPR-based diagnostic tests.

Sara Sawyer, a virologist at the University of Colorado, is trying to go one step further in the testing world. She's trying to develop a low-cost test people could use at home that would reveal whether they are infected days before they show any symptoms.

"For two years, we've been working on trying to develop a diagnostic that can pick up on the earliest stages of common respiratory diseases," Sawyer says. Her test doesn't look for the virus itself. Instead, it looks for a response to the virus by the cells of a person who is infected.

The idea is that once cells in the nose and throat are infected, certain genes are switched on that aren't normally switched on. Sawyer says it's possible to detect those "up-regulated" genes in saliva instead of the nasal swab other coronavirus tests rely on. The question is, can she distinguish the new coronavirus from other viruses. She thinks she can.

But do others agree?

"The answer is maybe," says Benjamin tenOever, a virologist at the Icahn School of Medicine at Mount Sinai in New York City. He says yes, infection by the virus that causes COVID-19 results in different genes being up-regulated, compared with flu or other viruses. He's just skeptical the technology exists to be able to detect those differences.

"I'd say theoretically it is possible," tenOever says. "She's a very smart scientist. And so if she says she can do it, I would give her the benefit of the doubt."

Sawyer has formed a company to build her test kit. If society is to reopen, she says, there will have to be easy ways for people to check their infection status. She's in the process of designing and raising money for a study to validate her test's accuracy.

"We think saliva is the key to moving these tests out of the doctor's office," Sawyer says, because all people would have to do to collect a sample is spit in a cup. No blood draws, no nasal swabs. Easy.

If it works.

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Next Generation Of COVID-19 Virus Tests Could Get Faster And Cheaper With CRISPR : Shots - Health News - NPR

CRISPR could speed growth and increase disease resistance in farmed fish, boosting aquaculture sustainability – Genetic Literacy Project

The potential of fish and shellfish production to feed a growing global population could be significantly enhanced through advances in genetics and biotechnology, researchers have said.

Many species of fish and shellfish have been domesticated relatively recently compared with most livestock species, and so have diverse gene pools with major potential for selective breeding, according to the review paper in Nature Reviews Genetics.

The development of tools to gain insight into the genetics of these species, and apply such tools for breeding and management, provides opportunities to release that potential, researchers say.

Most aquaculture species can produce many offspring, and large populations with improved genetics can be bred quickly for improved production performance.

The benefits may include improved growth, resistance to disease or robustness in diverse farming environments.

Farmed fish is on course to overcome wild fish as the main source of seafood, and consequently genetic tools and expertise are in high demand to increase the efficiency and sustainability of aquaculture systems, which currently rely mostly on unselected stocks.

Insight into the genomes of species can enable careful selection of a farming population with desirable traits, and monitoring genomic variation will help maintain genetic diversity as farm populations develop.

In the future, technologies such as genome editing could be used to introduce desirable traits, such as disease resistance, into farmed species, and surrogate breeding could be employed to support production of preferred species.

The review paper, collaboration between experts from Universities of Edinburgh, Exeter, Stirling, and Aberdeen, is an output of the AquaLeap consortium project.

AquaLeap is funded by the Biotechnology and Biological Sciences Research Council, the Natural Environment Research Council and the Scottish Aquaculture Innovation Centre, in partnership with the Centre for Environment, Fisheries and Aquaculture Science, Hendrix Genetics, Xelect, The National Lobster Hatchery, Tethys oysters, and Otter Ferry SeaFish.

Environmental Biologist Dr Eduarda Santos, from the University of Exeter and co-author of the study said: The rapid expansion of aquaculture has contributed to increased food security across the globe, however, issues related to domestication of desired species and emergence of diseases, limit its further development.

Genomics has the potential to offer solutions to many of these limitations by improving our knowledge of the genomes of cultured organisms, genetic selection, and better understanding of the dynamic interactions between genes and the environment, to maximise food production.

Dr Jamie Stevens, also from the University of Exeter and co-author added: We only have to look at the example of Atlantic salmon to see the immense value of a sequenced genome to the relatively recent optimisation of a wild species for the aquaculture market.

Similarly, we anticipate the delivery of a genome for other species, including the European lobster, will offer similar opportunities to develop molecular tools with which to rapidly increase the potential of lobster as an aquaculture species and improve the sustainability of its wild populations.

Professor Ross Houston, the Roslin Institute: There is a timely opportunity to harness the potential of farmed aquatic species, to ensure food security for a growing population. Genomic selection and biotechnology can speed up this process, and recent developments in these fields will soon be translated to benefit aquaculture production for many of these species across the world.

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The optimal immune repertoire for bacteria – Penn: Office of University Communications

Before CRISPR became a household name as a tool for gene editing, researchers had been studying this unique family of DNA sequences and its role in the bacterial immune response to viruses. The region of the bacterial genome known as the CRISPR cassette contains pieces of viral genomes, a genomic memory of previous infections. But what was surprising to researchers is that rather than storing remnants of every single virus encountered, bacteria only keep a small portion of what they could hold within their relatively large genomes.

Work published in the Proceedings of the National Academy of Sciences provides a new physical model that explains this phenomenon as a tradeoff between how much memory bacteria can keep versus how efficiently they can respond to new viral infections. Conducted by researchers at the American Physical Society, Max Planck Institute, University of Pennsylvania, and University of Toronto, the model found an optimal size for a bacterias immune repertoire and provides fundamental theoretical insights into how CRISPR works.

In recent years, CRISPR has become the go-to biotechnology platform, with the potential to transform medicine and bioengineering. In bacteria, CRISPR is a heritable and adaptive immune system that allows cells to fight viral infections: As bacteria come into contact with viruses, they acquire chunks of viral DNA called spacers that are incorporated into the bacterias genome. When the bacteria are attacked by a new virus, spacers are copied from the genome and linked onto molecular machines known as Cas proteins. If the attached sequence matches that of the viral invader, the Cas proteins will destroy the virus.

Bacteria have a different type of immune system than vertebrates, explains senior author Vijay Balasubramanian, but studying bacteria is an opportunity for researchers to learn more about the fundamentals of adaptive immunity. Bacteria are simpler, so if you want to understand the logic of immune systems, the way to do that would be in bacteria, he says. We may be able to understand the statistical principles of effective immunity within the broader question of how to organize an immune system.

Because of CRISPRs role in the bacterial immune response, the researchers were interested in developing a physical model that could describe the role of the CRISPR cassette during a viral infection. They were specifically interested in why bacteria tend to store only 50-100 viral DNA snippets, or spacers, from past infections when their genomes could easily hold thousands. The puzzle is that the bacteria go to the trouble of implementing this memory system, but they keep a shallow memory, says Balasubramanian. You would think that remembering more would be better.

As the researchers developed a mathematical model to look at bacterial survival, they could adjust the models parameters, such as the number of viruses the bacteria encountered and the number of spacers held within the genome, to see how these factors affect the bacterias overall chance of survival. They found that there was an optimal amount of memory that, surprisingly, only consisted of a few dozen spacers.

Why is having less memory more optimal? Memory is useless unless you have a way to use it, says Balasubramanian. This is because the spacers must be transcribed and attached onto the Cas proteins that mount an immune response, and there are only so many Cas proteins to go around. This means that there is an opportunity cost to keeping too much memory, which results in a trade-off between how much memory can be stored and how quickly bacteria can respond to a new infection. Cells are full of molecular machines, and all machines have constraints. Because that machinery is limited, bacteria only keep whats most useful, he says.

Another insight that was a key for their model was the need for multiple Cas protein recognitions of new viral infections. To prevent the bacteria from making mistakes, multiple Cas proteins are required to bind to and recognize a virus before mounting an immune response. By incorporating this requirement into the model, the researchers were able to understand the importance of limited resources, in this case Cas proteins, in determining the optimal amount of bacterial immune memory.

The researchers now plan to look at how other immune mechanisms affect how deep a bacterias memory should be. They also plan to study how bacteria use their relatively shallow memory to protect themselves from different types of viruses to see if, for example, bacteria keep more memory of viruses that are more dangerous or more common.

This work represents a unique, physics-based approach to study a biological mechanism that has become a widely used tool in biotechnology but still still remains poorly understood in terms of its natural function. As theorists, we think about the principles underlying function, says Balasubramanian. This is one of the first papers to try to establish the computational principles underlying CRISPR-based immunity, and it comes to an interesting conclusion.

Vijay Balasubramanian is the Cathy and Marc Lasry Professor in the Department of Physics and Astronomy in the School of Arts & Sciences at the University of Pennsylvania.

This research was supported by the Simons Foundation (Grant 400425) and National Science Foundation Center for the Physics of Biological Function (Grant PHY-1734030).

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Growing Application Areas of CRISPR Technology Market Driving Growth in Healthcare Sector – WhaTech Technology and Markets News

The CRISPR technology market is expected to grow from USD 562 million in 2018 to USD 1,715 million by 2023, at a CAGR of 25%.

The report"CRISPR Technology Marketby Product (Enzymes, Kits, gRNA, Libraries, Design Tools), Service (gRNA Design, Cell Line Engineering), Application (Biomedical, Agricultural), End User (Pharma & Biopharma Companies, Academics, CROs) - Global Forecast to 2023", The CRISPR technology market is expected to grow from USD 562 million in 2018 to USD 1,715 million by 2023, at a CAGR of 25% during the forecast period. The major factors driving the CRISPR technology market include the rising funding from government and private organizations and the high adoption of CRISPR technology.

Browse132 market data Tables and24 Figures spread through163 Pages and in-depth TOC on"CRISPR Technology Market"

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The CRISPR products segment is expected to command the largest share of the CRISPR technology market during the forecast period.

The CRISPR technology market, by product and service, is estimated to be dominated by the products segment in 2018. This is attributed to the fact that the CRISPR technology is being adopted quickly by academics and researchers, pharma and biotech companies.

The enzymes segment is expected to account for the largest share of the products market, being one of the key ingredients in the CRISPR process. Companies like Merck KGaA and Thermo Fisher Scientific are providing hands-on training to researchers, which will increase the demand for CRISPR products in the future.

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Biomedical applications to occupy the majority of the market, by application, and grow at the fastest rate during the forecast period.

The biomedical applications segment is projected to be the fastest-growing segment of the market, by application, during the forecast period. Developments in gene therapy, drug discovery, and diagnostics, due to the application of CRISPR, are driving the growth of this biomedical segment.

Many companies have also invested in drug discovery and gene therapy companies that are using CRISPR technology.

North America is projected to account for the largest share of the CRISPR technology market, by region, during the forecast period.

North America is estimated to account for the largest share of the market in 2018. The large share of CRISPR technology in this region is majorly attributed to the rising government and private funding, presence of major pharma and gene therapy companies, and the adoption of CRISPR in a number of applications.

Crops that are treated with CRISPR-based gene editing are not considered as GMOs in the US, which has attracted the attention of agricultural companies to the commercialization of CRISPR-edited crops.

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Leading Companies

Prominent players in the CRISPR Technology market are Cellecta, Inc. (US), Thermo Fisher (US), GeneCopoeia, Inc.

(US), Applied StemCell (US), Synthego Corporation (US), OriGene Technologies (US), Horizon Discov

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First patient of a landmark clinical trial to treat a genetic eye disorder with CRISPR gene therapy receives treatment – Massive Science

We are currently living in a situation of extreme uncertainty, and if you are like me, you may have noticed yourself feeling extra anxious lately. Maybe you feel a constant ache it in your shoulders and neck. Maybe you are compulsively checking your phone, unable to tear your eyes away from Twitter and Facebook, or maybe you're extra irritable. According to medical professionals, these are very normal responses to the coronavirus pandemic.

Luckily there are lots of things you can do on your own to help ease the stress. Here are a few that work for me personally (note: I am not a medical professional). Not all of these will work for everyone, so don't beat yourself up if you try something to lessen your anxiety and it doesn't do much. Each of us is unique in our experiences and reactions to stressors!

Create something: Make something with your hands. You can cook or bake, put together a puzzle, color or draw, work in your garden or yard (if you have one), or even clean out your car. Whatever you choose, try to really focus on what you are doing instead of letting your mind wander. Don't worry about making something perfect just enjoy the process!

Go outside or get moving inside: Unless you are currently under lockdown, and assuming you stay at least 6 feet from others, it is safe to go outside. Exercise can help you redirect nervous energy. It also gets your feel-good neurotransmitters flowing. By the way, dancing in your living room counts as exercise!

Step away from your phone: Put the phone down. Leave it in another room while go about your other activities. It will feel weird, but I promise you that logging off Twitter and other social media for half an hour will not harm you. To be clear, your phone isn't the root cause of your anxiety, but a constant barrage of COVID-19 related news isn't helpful, either.

Give yourself a break: If you are really feeling anxious and it is keeping you from your daily activities, try just letting yourself be. A lot of times the pressure we put on ourselves to stay productive, keep working, clean the house, and so on keeps us paralyzed. Banish the word "should" from your vocabulary for now, and just do the best you can. Sometimes just giving yourself permission to slack off is enough to get your motivation and focus back.

Try mindfulness: Mindfulness seems like the hip, hot thing to do lately, but there's a reason for that it works. There are tons of online resources and apps for learning mindfulness. I am most familiar with Headspace, and the thing I like most about it is that students (including grad students!) can get access to the full app for $10/year (usually $70). There is a lot of material in the app, and in my opinion it's worth it.

If full-on mindfulness isn't for you, but you need a way to stay calm when it feels like the world is falling apart around you, the 54321 method of grounding yourself is a good place to start. Take a deep breath, then look around you for five things that stick out to you in the moment, and say them out loud. Then repeat that with four things you can feel, three sounds you hear, two things you can smell, and one thing you can taste. Then take another deep breath.

We're all in this together. While you may have to stay physically distant from people right now, don't forget to connect socially in any way you can. And if you are feeling totally overwhelmed or depressed, please reach out to a mental health professional.

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First patient of a landmark clinical trial to treat a genetic eye disorder with CRISPR gene therapy receives treatment - Massive Science

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