Archive for the ‘Crispr’ Category

A company developing means to edit genes at the single-letter level has raised nearly $200 million in its initial public offering.

Cambridge, Massachusetts-based Beam Therapeutics said Wednesday evening that it had priced its IPO at $180 million, or $17 per share. The company began trading Thursday on the Nasdaq under the ticker symbol BEAM.

The company had filed to go public in September, aiming at a $100 million IPO. J.P. Morgan, Jeffereies and Barclays acted as joint book runners.

Its last major financing before the IPO was a Series B funding round that it raised in March worth $135 million. The company launched with an $87 million Series A round in May 2018 after operating in stealth mode for a year.

Beams work focuses on base editing, a technology that enables editing of the genome letter by letter, as opposed to the cutting technique usually involved in CRISPR/Cas9 gene editing. Each letter of the genome corresponds with a specific nucleobase: A for adenine, G for guanine, T for thymine and C for cytosine. In other words, base editing would enable changing C to T, G to A and so forth. The company has a licensing agreement with the Massachusetts Institute of Technology and Harvard Universitys Broad Institute for co-founder and CRISPR/Cas9 pioneer Feng Zhangs RNA base-editing technology.

Its most recent presentation of data was in April 2019, when it showed preclinical data from its base editing platform at the American Society of Gene and Cell Therapys annual meeting in Washington. For the study, Beam used the base editor BE4 for multiplex base editing of engineered CAR-T cells, showing the ability to knock out expression of three cell surface targets TRAC, B2M and PD-1 in 95%, 95% and 88% of cells, respectively. That technique, the company said, may be able to create CAR-T cells with improved therapeutic properties.

Gene editing is a key technology used for the creation of CAR-T cells derived from donors rather than from patients own T cells, also known as allogeneic or off-the-shelf CAR-Ts, with both CRISPR/Cas9 and TALEN gene-editing technology being used. Without such modifications, off-the-shelf CAR-T cells would be rejected by the body.

Photo: Spencer Platt, Getty Images

Original post:
Beam Therapeutics raises $180M in IPO - MedCity News

By Michael Le Page

Vchal/Getty Images

CRISPR gene-edited immune cells have been injected into threepeople with advanced cancer without any serious side effects, the first trial of its kind in the US. It is also the first CRISPR cancer trial in the world to publish its findings, and the encouraging results will pave the way for many more trials.

Its an important milestone, says Waseem Qasim at theUCLGreat Ormond Street Institute of Child Health in the UK, who is carrying out a similar trial there.

The US trial was intended only to assess safety. The threeparticipants had tumours that hadnt responded to other treatments, and were given only one dose of gene-edited cells.

Advertisement

Is it safe and feasible? says team member Edward Stadtmauer at the University of Pennsylvania. I think thats what we demonstrated.

Many cancers involving blood cells are now treated by removing immune cells from individuals, adding a gene that makes them target cancer cells and putting them back in the body.

But this treatment doesnt work for everyone, says Stadtmauer. And in some, it works at first but they later relapse.

The hope is that using gene editing to delete genes in addition to adding the targeting gene will make this approach even more effective. For instance, immune cells have a safety switch, called PD-1, that other cells can flip to say dont hurt me. Many cancers exploit this to avoid immune attacks.

In this trial, the team removed immune cells from three peoplewho hadtumours with the same protein on their surface. A virus was used to add a gene to make the immune cells target this protein.

Next, three genes, including PD-1, were deleted using CRISPR. After six weeks, the cells were put back in the individuals, where they survived for at least 9 months.

There were two big safety concerns. Firstly, CRISPR can cause unintended changes to genomes that could turn cells cancerous. Deleting three genes means cutting around each one in three spots in the genome, for instance, and the wrong ends can be joined up. This did happen in some cells, but there was no sign of any turning cancerous.

The other worry was that lingering traces of the CRISPR protein used for gene editing might trigger an immune reaction, since it is a bacterial protein. There was no sign of this.

The trial wont continue because its 2016 gene-editing technology is already outdated, says Carl June, also at the University of Pennsylvania. In particular, a new form of CRISPR called base editing can be used to inactivate genes without cutting DNA, which should reduce the cancer risk even further. We are very attracted to that, says June.

There are also many other ways to edit immune cells to make them more effective, says Stadtmauer. The possibilities are limitless based on our imagination and scientific focus.

In particular, Stadtmauer wants to create off-the-shelf cells that could be given to any patient, rather than modifying each patients own cells. This would speed up treatments and greatly reduce costs.

Qasims team has already saved lives in an ongoing trial at Great Ormond Street using off-the-shelf cells created by an older form of gene editing called TALEN. But these cells have to be given as part of a drastic treatment, which is followed by a bone marrow transplant that kills off the edited cells. Stadtmauer wants to create cells that can survive indefinitely in peoples bodies.

The risk of edited cells turning cancerous or starting to attack healthy cells would be higher if they survive longer. But it is also possible to add a self-destruct mechanism triggered by a specific drug to kill them off if necessary.

There have been a number of trials of CRISPR-edited immune cells for treating cancer in China, but no results have yet been published.

Journal reference: Science, DOI: 10.1126/science.aba7365

More on these topics:

See the original post here:
CRISPR cancer trial finds that gene-edited immune cells are safe - New Scientist News

Spurred by promising clinical results in an important trial, each of the three major CRISPR stocks had a great performance in the second half of 2019. Unfortunately, they didn't keep the momentum going in the first month of 2020.

Shares of Intellia Therapeutics (NASDAQ:NTLA) fell 18.8% in January, according to data provided by S&P Global Market Intelligence. That was followed by a 14.7% loss for shares of CRISPR Therapeutics (NASDAQ:CRSP) and a 10.7% tumble for shares of Editas Medicine (NASDAQ:EDIT).

While each has recovered some ground in the first week of February, this trio of pharma stocks is no stranger to volatility. Investors should probably expect that to continue as clinical programs advance in 2020.

Image source: Getty Images.

In November, CRISPR Therapeutics reported data for the first two individuals in the trial, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), treated with its lead drug candidate CTX001. Both enjoyed significant benefits in their standard of living, which investors interpreted as a sign that CRISPR gene editing might actually live up to the hype.

That fueled annual gains of 113% for CRISPR Therapeutics last year. While Editas Medicine and Intellia Therapeutics gained only 30% and 7%, respectively, each had been sitting at a year-to-date loss in October.

What relevance does that have for the tumbles taken in January? First, it's not unusual for stocks to regress to the mean. Stocks that are red hot eventually cool off, while those that tumble without good reason eventually recover some ground.

Second, and the more important consideration for investors, is that the early stage results for CTX001 mean relatively little for the industry's pipeline of CRISPR-based gene editing drug candidates.

Consider that CTX001 is an ex vivo tool. Researchers harvest bone marrow from patients, extract specific types of stem cells, and engineer those with CTX001. The engineered stem cells are then grown in the lab before being reinjected into the patient.

Many other CRISPR-based drug candidates are designed as in vivo tools. That means the gene editing payloads are designed to engineer a patient's DNA while inside the body. An in vivo approach is inherently more complex and will be more difficult to control compared to an ex vivo approach.

Put another way, investors cannot take the promising, early stage results from CTX001 and extrapolate it broadly across all first-generation CRISPR tools. Wall Street certainly isn't, if the correlation between technical approach and stock performance is any guide.

Consider that the two most advanced drug candidates from CRISPR Therapeutics rely on ex vivo engineering. By contrast, the lead drug candidate from Editas Medicine relies on in vivo methods.

The lead pipeline asset from Intellia Therapeutics is also an in vivo tool, though unlike the lead assets from its peers, it has yet to advance to clinical trials.

Investors should expect 2020 to be a busy year for these CRISPR stocks. CRISPR Therapeutics will have more clinical data from CTX001 and the first set of data for its lead oncology asset CTX110.

Similarly, Editas Medicine should have results for EDIT101 and progress additional assets, while Intellia Therapeutics is preparing to finally enter the clinic with NTLA-2001 in the second half of the year.

Investors cannot know if the next batch of results will be as rosy as the initial data for CTX001, but they can probably expect another year of volatile stock movements.

Go here to read the rest:
Here's Why CRISPR Stocks Fell in January - The Motley Fool

The first targets of trials in humans are serious diseases we know are caused by a single gene, but as we understand more about combinations of genes increasing risk factors for other diseases, Crispr could be used more widely

BL PREMIUM

01 February 2020 - 06:17 Hannah Kuchler

Jennifer Doudna wears her responsibility lightly. The scientist who co-discovered Crispr (clustered regularly interspaced short palindromic repeats) does not appear to be weighed down by the burden of her creation: the revolutionary gene-editing technology that promises to empower humans to control our own genome. She waves and smiles as she bypasses the hostess stand at Gather, an organic, farm-to-table restaurant in Berkeley. Its the sort of place that wears its principles on its sleeve a fitting venue for a discussion of the ethical conundrums that Crispr has unleashed.

Doudna appears to have thrown on her blazer in a rush, squashing down one side of her shirt collar. Since news of her scientific breakthrough was published in 2012, she has learnt to toggle between the white coat of her lab work, building on that initial discovery, and her suited-and-booted role pushing politicians and lawmakers to contemplate the consequences of changing the human genome.

Continued here:
LUNCH WITH THE FT: Jennifer Doudna, scientist and gene editor at the heart of the Crispr maelstrom - Business Day

The recent performance of Fastly (NYSE:FSLY) stock in the market spoke loud and clear to investors as FSLY saw more than 1.51M shares in trading volumes in the last trading session, way higher than the average trading volume of 1.51M shares by far recorded in the movement of Fastly (FSLY). At the time the stock opened at the value of $22.60, making it a high for the given period, the value of the stock jumped by 1.43%. After the increase, FSLY touched a low price of $22.10, calling it a day with a closing price of $22.43, which means that the price of FSLY went 22.75 below the opening price on the mentioned day.

Given the most recent momentum in the market in the price movement of FSLY stock, some strong opinions on the matter of investing in the companys stock started to take shape, which is how analysts are predicting an estimated price of $26.11 for FSLY within consensus. The estimated price would demand a set of gains in total of 8.5%, which goes higher than the most recent closing price, indicating that the stock is in for bullish trends. Other indicators are hinting that the stock could reach an outstanding figure in the market share, which is currently set at 38.62M in the public float and 2.21B US dollars in market capitalization.

When it comes to the technical analysis of FSLY stock, there are more than several important indicators on the companys success in the market, one of those being the Relative Strength Indicator (RSI), which can show, just as Stochastic measures, what is going on with the value of the stock beneath the data. This value may also indicate that the stock will go sideways rather than up or down, also indicating that the price could stay where it is for quite some time. When it comes to Stochastic reading, FSLY stock are showing 19.29% in results, indicating that the stock is neither overbought or oversold at the moment, providing it with a neutral within Stochastic reading as well. Additionally, FSLY with the present state of 200 MA appear to be indicating bullish trends within the movement of the stock in the market. While other metrics within the technical analysis are due to provide an outline into the value of FSLY, the general sentiment in the market is inclined toward positive trends.

With the previous 100-day trading volume average of 964502 shares, CRISPR Therapeutics AG (CRSP) recorded a trading volume of 902880 shares, as the stock started the trading session at the value of $52.71, in the end touching the price of $53.48 after jumping by 1.46%.

CRSP stock seem to be going ahead the lowest price in the last 52 weeks with the latest change of 82.28%.Then price of CRSP also went backward in oppose to its average movements recorded in the previous 20 days. The price volatility of CRSP stock during the period of the last months recorded 4.09%, whilst it changed for the week, now showing 3.21% of volatility in the last seven days. The trading distance for this period is set at -6.90% and is presently away from its moving average by -14.88% in the last 50 days. During the period of the last 5 days, CRSP stock lost around -3.36% of its value, now recording a sink by 8.63% reaching an average $49.32 in the period of the last 200 days.During the period of the last 12 months, CRISPR Therapeutics AG (CRSP) dropped by -12.19%.

According to the Barcharts scale, the companys consensus rating fall to 4.27 from 4.40, showing an overall improvement during the course of a single month. Based on the latest results, analysts are suggesting that the target price for CRSP stock should be $53.48 per share in the course of the next 12 months. To achieve the target price as suggested by analysts, CRSP should have a spike by 0% in oppose to its present value in the market. Additionally, the current price showcases a discount of 48.58% when compared to the high consensus price target predicted by analysts.

CRSP shares recorded a trading volume of 857794 shares, compared to the volume of 1.21M shares before the last close, presented as its trading average. With the approaching 3.21% during the last seven days, the volatility of CRSP stock remained at 4.09%. During the last trading session, the lost value that CRSP stock recorded was set at the price of $53.48, while the lowest value in the last 52 weeks was set at $29.34. The recovery of the stock in the market has notably added 82.28% of gains since its low value, also recording -10.03% in the period of the last 1 month.

Read more:
Stocks That Are Finally Turning The Corner: Fastly (FSLY), CRISPR Therapeutics AG (CRSP) - US Post News

The UK must break away from Europes restrictive agricultural gene-editing rules, science writer Matt Ridley told the UKs House of Lords on January 30. A vocal advocate for technological innovation, Ridely argued that Britain should embrace CRISPR and other new breeding techniques, or risk falling behind enterprising nations, including the US, Canada and Argentina, that have recognized the technologys benefits.

While critics warn that gene-edited crops and animals could pose a risk to human health and the environment, Ridely countered that no data support such concerns and offered several examples of how Britain stands to gain by taking an evidence-based position on CRISPR:

There is no clearer case of a technology in which we could and should take the lead, but in which we are and will be held back if we do not break free from the EU approach. That would not be a race to the bottom but the very opposite: a race to the top.

For example, if we allowed the genome-edited blight-resistant potatoes developed at the Sainsbury Laboratory to be grown here in the UK, we would be able to greatly reduce the spraying of fungicides on potato fields, which happens up to 15 times a year, harming biodiversity and causing lots of emissions from tractors. That would be an improvement, not a regression, in environmental terms.

See the original post here:
Video: House of Lords member and science writer Matt Ridely urges UK to 'break free' of Europe's restrictive CRISPR crop rules - Genetic Literacy...

SEOUL, South Korea, Feb. 3, 2020 /PRNewswire/ --Scientists at Dongguk University successfully treated Alzheimer's disease in mice using the gene editing tool CRISPR-Cas9. They used the tool to edit out the Alzheimer's causing gene in the mice, thereby improving their memory and other cognitive functions. While this is only a first step and much research remains to be done, these promising results put gene therapy on the map of possible treatments for Alzheimer's.

As Alzheimer's disease continues to elude the efforts of modern medicine, scientists turn to gene editing technology to treat, or 'manage' it. Alzheimer's is commonly associated with the BACE 1 gene, which drives the production of amyloid-proteins in the brain. In the brains of Alzheimer's patients, this protein accumulates on the outsides of neurons or nerve cells as "plaque," and is thought to be one of the main causes of the disease. Using "DNA scissors" called CRISPR-Cas9, which can cut out a specified part of a DNA sequence, Dr. Jongpil Kim and his colleagues from Korea (Dongguk University) and the USA attempted to edit out the BACE 1 gene in the neurons of the adult mouse brain.

Theirs is the first in vivo attempt (using cells inside living mice) to test the effectiveness of CRISPR-Cas9 in stopping Alzheimer's progression. Their ultimate ambitionlike that of many others in the fieldis to develop treatments for different forms of dementia, which would dramatically improve patients' quality of life. "We aimed to see whether CRISPR-Cas9, one of the latest developments in biotechnology, can open up a new direction for treating dementia, which is, at present, considered an incurable disease," Dr. Kim says.

Their results indeed look promising. Within 8 to 12 weeks of treatment with the CRISPR-Cas9 technology, the plaque surrounding affected neurons in the mice dramatically decreased and their cognitive functions improved substantially. The mice also showed better associative learning and spatial working memory. Further, there was no evidence of increasing mutations in other, non-targeted parts of the genome, suggesting minimal side-effects.

All this indicates the huge potential of gene editing technologies to treat neurodegenerative diseases: by removing faulty genes at their source, disease progression can be halted, or even reversed, in just a few weeks.

The authors advise caution, however, as gene editing cannot be undone. In Dr. Kim's words, one must ensure that "no detrimental, and potentially very rare, genomic alterations are caused". Thus, this technique requires much more research before it can be applied to human subjects.

Nonetheless, as another remark by Dr. Kim goes, "this study shows how CRISPR-Cas9 can be applied to the treatment of neurodegenerative diseases as well."

Reference

Author:

Jongpil Kim

Title of original paper:

In vivo neuronal gene editing via CRISPRCas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease

Journal:

Nature Neuroscience

DOI:

10.1038/s41593-019-0352-0

Affiliations:

Departments of Chemistry and Biomedical Engineering, Dongguk University

Media contact:Jongpil Kim 233458@email4pr.com+82-10-4013-3685

SOURCE Dongguk University

Read more from the original source:
DNA "Scissors" Could Cut Out the Alzheimer's Causing Gene in Mice - PRNewswire

How Mammoth Biosciences is Using Protein Discovery to Unlock the Potential of CRISPR

Its happening again. The world looks on anxiously as new updates emerge almost hourly from China. Just this morning I read in my NY Times Daily Briefing that the death toll from a new coronavirus originating in Wuhan, China, has reached 170, with 7700 people sickened worldwide from the virus. People are asking, is this the next pandemic?

We worry about pandemics because we worry about our ability to stop them and to save lives. Every time a new pandemic, or potential pandemic, emerges, we are reminded that we still dont have an affordable, accessible, rapid and accurate molecular diagnostic test that can be used in the field. Were such a diagnostic to exist, we could more quickly contain the spread of diseases like the new coronavirus, or Ebola, which has killed hundreds of thousands in Africa, or any other disease. It could literally change everything.

The reality is we will continue to see pandemics. Our goal is to build a disposable, portable, ultra-accurate test, similar to a pregnancy test format but molecular, says Trevor Martin, co-founder and CEO of Silicon Valley startup Mammoth Biosciences.

His company is closer to that goal than you might think and theyre doing it with CRISPR.

When you think of CRISPR, you most likely think of gene editing. Youve probably read a dozen articles touting the potential for these molecular scissors to cure diseases such as blindness or cancer. Indeed, clinical trials that began last year have already led to groundbreaking outcomes. But CRISPR can and should be so much more than that.

CRISPR is a microbial immune system, the way by which bacteria fight off viruses, which act by injecting viral DNA into the host genome. And while Cas9 the molecular scissors may be the star of CRISPR, the CRISPR system is actually comprised of several Cas protein families, each of which has its own unique functions. This was this premise upon which Mammoth Biosciences was built: using deep protein discovery focused on CRISPR proteins, identify new proteins and unlock new functions. Its an approach that has paid off.

Our thesis is that novel functions unlock new products, says Martin. From the very beginning the founding of the company [CRISPR] proteins with novel trans-cleavage ability created diagnostics, and that invented this brand new field (CRISPR diagnostics) that people didnt think could exist.

Using CRISPR as a diagnostic tool rather than an editing tool may have been a surprise when Mammoth introduced it to the world two years ago, but Martin says that if we think about CRISPR as a homing beacon instead of just as molecular scissors, the diagnostic applications arent so surprising after all.

[CRISPR] is really a homing beacon, and you can use it to home in on something and then report on it, home in on it and cut it, home in on it and edit a base, activate a gene, destroy a sequence, whatever you want to do. And once you start thinking about it that way, it becomes more obvious whats going on. Diagnostics becomes more clear, but so do other next-generation therapeutic applications, he says.

Mammoths goal is to build out a platform for next-generation CRISPR-based diagnostics and therapeutics. By continuing to search the protein space deeply for new CRISPR proteins with new functions, the company is building a comprehensive toolbox that researchers can use to create new therapeutics and improved diagnostics tools. Their toolbox already contains proteins from the Cas12, Cas13, and Cas14 protein families and it is Cas14 that is especially interesting.

CRISPR proteins can actually be quite large Cas9, for example, is so big that it is difficult to deliver using adenovirus (one of the most common delivery methods for gene editing). The size issue is compounded when considering CRISPR add-ons (like CRISPR prime), which expand upon the scissor function of Cas9 to add precise capabilities like changing one base for another, adding a gene instead of removing a gene, and directed genome targeting. Each add-on makes the CRISPR complex even bigger and harder to deliver.

But Cas14 is unique. It comes from Archaea a close relative to bacteria and its much smaller than other CRISPR proteins like Cas9. It can easily be delivered via adenovirus, it can accommodate CRISPR add-on protein domains, and its small size even makes it effective for delivery using lipid nanoparticles (LNPs). Martin thinks Cas14 can help the field reach the holy grail goal of in vivo gene editing, replacing the current system of removing cells from a patients body, editing them ex vivo in the laboratory, and then re-introducing the edited cells back into the patient.

Cas14 is also the poster child for Mammoths approach to use deep protein discovery for finding novel proteins with novel functions. It is one of the most diverse if not the most diverse of the CRISPR proteins weve discovered so far.

There are potentially many hundreds of [Cas14] proteins, says Martin, which means a ton of functionality that can be uncovered.

And, for some applications, Cas14 can target anywhere in the genome you want it to and has less off-target effects that have led some to worry about the risk to benefit ratio of using CRISPR to treat disease.

Of course, Cas14 is just a start, and its unlikely to be the right tool for everything. Different Cas proteins will be necessary for different applications, and in addition to Mammoths internal work, its their goal to partner deeply with companies to develop their CRISPR proteins and bring them into diagnostic and therapeutic products such as Martins dream rapid diagnostic field test. Mammoth is partnering with UCSF researchers on a coronavirus diagnostic, a partnership that couldnt have come too soon. And, the company recently announced a collaboration with Horizon Discovery to create a new generation of Chinese hamster ovary (CHO) cell line editing tools.

We are not experts in hundreds of disease areas by ourselves but we are the worlds best at developing proteins with these novel functionalities. Like [we did] with CRISPR diagnostics, were now building a platform on the therapeutic side, including working closely with companies with specific expertise to bring products to market, says Martin.

A recent injection of USD $45 million from investors with deep healthcare interests, such as Decheng, Mayfield, Verily, and Brook Byers, will enable the company to double down on their efforts to develop their toolbox of next-generation CRISPR diagnostics and therapeutics. And, recent executive hires bring significant industry expertise to the table from new CBO Peter Nell (co-founder of Casebia, a joint venture between Bayer and CRISPR Therapeutics) and new COO Ted Tisch (a former Synthego executive).

All too often it seems like we arent quite there yet when it comes to the promise of biotechnology. But when it comes to CRISPR, we really are almost there. With Mammoth Biosciences leading the way by providing the next-generation tools needed to fully realize the therapeutic and diagnostic potential of CRISPR, things might turn out a bit different the next time a pandemic rolls around.

Read more from the original source:
The CRISPR Platform for Next-Generation Therapeutics and Diagnostics - SynBioBeta

Written By: Dr. Ananya MukherjeeDepartment of Plant Science and innovationUniversity of Nebraska-Lincoln, USAPhoto Credit: Shutterstock

CRISPR is something you must have come across in your science news feed in the last couple of years or so. Even if you have not, you may have seen the big news that took social media by storm the genome-edited babies in China, Lulu and Nana. One might wonder what is this fuss all about and why is this technique in the news all of a sudden? To make sense of it all its important to delve a little into the history of CRISPR and why it became bigger than any other editing strategies that have been around for a while.

The scientists quest of genome or gene editing is not anything new. For several decades we have been striving to find techniques which will efficiently target genes of interest and make the changes we want them to make but with minimal off-targets. The reason why CRISPR is preferred over other tools is its efficiency, ease of manipulation and most importantly precision. Nearly 33 years ago Francisco Mojica from the University of Alicante, Spain found what we now know as CRISPR or Clusters of Regularly Interspaced Short Palindromic Repeats in E. Coli. Soon after that such regions of repeat DNA were found in another domain Archea as well. However, since then no other organism domain has shown CRISPR like repeats. Although the very fact that these unusual repeat sequences showed up or are conserved in two domains, making them the most widely distributed repeat sequences in prokaryotes. Its hypothesized that these repeats which are part of the bacterial immune system came about to fight the selective pressure created by viruses. In 2005 Alexander Bolotin, French National Institute for Agricultural Research found what we now know as Cas9, a helper enzyme of CRISPR that acts like molecular scissors to make specific edits in DNA. The beauty of CRISPR perhaps is in the fact that the gene we wish to edit doesnt need to be paired with enzymes like in other genetic methods, but Cas9 can be led to the gene via something called a guide RNA.

Soon after in 2007, CRISPR was used for S.thermophilus, which is used in the dairy and yoghurt industry, to resist bacteriophage attacks. Ever since its discovery, there has been no looking back for CRISPR. From agricultural applications like creating genetically edited crops tolerant to drought, crop diseases and higher yield to in-vitro human cells and animal models showing its applications in curing or reversing genetic defects like cystic fibrosis. In a recent article, National Geographic interviewed scientist Zachary Lipman from Cold Spring Harbor who is trying to put in fields high-yield tomatoes. This is not very surprising because since 2013 disease-resistant varieties of tomato are being developed by scientists, showing how far this technique has come. Even abiotic stress such as chill which tomato is very susceptible to, has been tackled with CRISPR by the introduction of genes that can resist cold weather. Essentially it comes down to knowing which gene or group of genes to target so that off-target effect is minimal and the right objective is achieved. Aside from tomato, strawberry, banana, grape, apple, watermelon, and kiwifruit are some more fruits which have been edited by CRISPR. Complex and large genomes such as date palm are now being targeted to be edited by CRISPR to tackle similar growth-related issues in a changing climate. Food crops are being manipulated with the help of CRISPR to produce everything from low gluten wheat to virus-resistant cacao in West Africa that can produce more chocolate. Mushrooms which are known for spoiling easily are being edited to have a longer shelf life. China has scientists working on improving yields in rice. Its no secret that with the boom in population and a changing climate the world is soon going to be in a massive struggle to have enough food for every mouth. With such attempts on a staple food crop, we can benefit in the right direction. These are just some exciting examples. There are lists of agriculture benefits that we have gotten from CRISPR and most of these crops are under varying stages of success.

Gene drives is another new application of CRISPR where a certain favourable trait can be passed down from parent to the next generation and thereby propagate in the entire population such as disease resistance or in agriculture- herbicide or pesticide resistance. In October of this year, David Liu of the Broad Institute of MIT and Harvard and postdoctoral fellow Dr. Andrew Anzalone came up with prime editing, which is a new form of CRISPR Cas9. The beauty of this technique stems from its ability to change the 4 bases of DNA A.T, G and C into any other base as deemed fit. Previous techniques were not as flexible and could not theoretically cure diseases like sickle cell anaemia which requires a specific A to T mutation to correct it. As of now several human trials for CRISPR to treat the disease have been approved and are all on the way. These include sickle cell anaemia to replace the lethal mutation, T cell editing to treat various types of cancer, photoreceptor cell editing to treat Night Blindness and many others. Hence almost every biological system has been affected by the development of CRISPR.

This now brings us to the big CRISPR story which had the world in the crossfire of several debates regarding the ethical concerns of such a fast developing and precise technique like CRISPR. In June 2017 a couple visited Southern University of Science and Technology in Shenzhen China to meet He Jiankui. He, a biophysicist, was attempting to edit an embryo to remove the surface protein CCR5 that HIV uses for establishing infection. In November 2018, twins Lulu and Nana were born with edited genomes and it was known that another woman was also pregnant with a third CRISPR baby. Despite public outrage, He has not admitted to any wrongdoing and has even published a video promotion of the project. This is mainly because sometime in 2017 the US National academy of sciences decided that human trials if strictly regulated are permissible and its up to the governments to decide what can and cannot be done, as reported in Science. Of course, there has been public criticism by Nobel laureates like David Baltimore but there are also scientists who have emailed words of praise to He. He since his controversial appearance in the Hong Kong summit of genome editing in 2018, has been believed to be under house arrest. He was let go from his university position and not made any public appearances since then.

On the face of it, such an edit seems to be a good step in the right direction. After all, any future parent would want his or her kid to be free from certain deadly disease like HIV. The main cause for concerns is the fact that the exact effect of the mutations induced are not known and CRISPR, despite being a sophisticated technique, does have off-target effects. There is no telling if some cells had started to divide in the embryo before the edit was introduced and a mosaic effect may happen in such a case. Taking into consideration all of these dangers, its hard to say if the attempt made was a success or not. However, this is a very slippery slope because designer babies may soon become the future. CRISPR is not without its off-targets and such off-targets in humans can have deadly consequences and may even be inherited as mutations by generations. Editing embryos may not be the best way to eradicate HIV which is more prevalent in the continent of Africa. Editing babies perhaps would be able to slow down HIV in 30 years but by then more techniques will come up to stabilize the spread anyway. There is also the fact that if humans start doing away with all diseases the population boom may be too much for the world to handle climate change and food shortage breathing down our necks. As of now, CRISPR has only grown stronger with a new report showing it can cut and splice whole chromosomes. In the University of Pennsylvania in Philadelphia clinical trials to treat cancer cells have been approved and are underway. The scientific community has no doubt made great strides to use this gene-editing technique positively but only time will tell if the progress of CRISPR continues an upward trajectory both scientifically and ethically.

See the original post:
The CRISPR Story : From Boon to Bane? - Multidimension Magazine

Top research study on Global Crispr And Crispr-Associated (Cas) Genes Market is an extensive compilation of innovative developments, growth opportunities and revenue analysis of top-tier Crispr And Crispr-Associated (Cas) Genes Industry aspirants. The report states the growth trajectory of Global Crispr And Crispr-Associated (Cas) Genes Market growth during 2020-2026. Key industry aspects like SWOT analysis, Porters five forces analysis, and market statistics are mentioned. Global Crispr And Crispr-Associated (Cas) Genes Industry is expected to reach xx million USD in 2020 and will grow at a CAGR of xx% during 2020-2026.

Know About Crispr And Crispr-Associated (Cas) Genes Market Research Report

The prime manufacturers of Crispr And Crispr-Associated (Cas) Genes Market is as follows:

Mirus Bio LLCAddgeneGE Healthcare DharmaconCaribou BiosciencesTakara Bio USAHorizon Discovery GroupCRISPR THERAPEUTICSThermo Fisher ScientificMerck KGaAEditas MedicineIntellia Therapeutics

The production, regional trade, investment opportunities, mergers & acquisitions and sales channels of Crispr And Crispr-Associated (Cas) Genes Industry are stated. The top manufacturers, product types, applications, and market share is stated. The regional Crispr And Crispr-Associated (Cas) Genes analysis covers North America, Europe, China, Japan, India, South America, Middle East, and Africa and the rest of the world.

Global Crispr And Crispr-Associated (Cas) Genes Research Report offers complete details about industry chain structure, raw materials, pricing analysis, company profiles, and product specifications. The sales analysis, value chain optimization, strategic insights on Crispr And Crispr-Associated (Cas) Genes Industry, product launches and market risks are mentioned in this report. The country-level analysis of Crispr And Crispr-Associated (Cas) Genes Report covers USA, Canada, Mexico, Germany, France, UK, Russia, Italy, China, Japan, India, Korea, Australia, Brazil, Argentina, Colombia, Saudi Arabia, UAE, Egypt, South Africa and rest of the world. Market scope, revenue, information on product services and gross margin status is covered in this report. The import-export scenario, demand-supply, consumer behavior, and complete details on distributors, suppliers, traders, and dealers in Crispr And Crispr-Associated (Cas) Genes Market are stated.

Know More About This Report Or Request Free Sample With Custom Queries (If Any): https://reportscheck.biz/report/43984/global-crispr-and-crispr-associated-cas-genes-industry-market-research-report/

Key Extracts From Table Of Content:

Section 1: Market Introduction and Overview

Section 2: Product Overview, Classification, Scope

Section 3: Competitive Crispr And Crispr-Associated (Cas) Genes Market scenario based on Top Manufacturers

Section 4: Historic Study of Crispr And Crispr-Associated (Cas) Genes Market Based on Region, Type, Application

Section 5: Company Profiles of Key Crispr And Crispr-Associated (Cas) Genes Players, Market Share, Product Portfolio and Regional Presence

Section 6: Manufacturing Cost Analysis, Key Business Figures, Gross Margin, SWOT Analysis

Section 7: Regional Analysis, Market Status and Prospect From 2015-2026

Section 8: Market Dynamics, Marketing and Sales Channels, Distributors and Customer Analysis

Section 9: Financial Highlights of Crispr And Crispr-Associated (Cas) Genes Market Including Total Revenue, Products, Services, Opportunities, and Market Risk Analysis

Section 10: Global Crispr And Crispr-Associated (Cas) Genes Market Forecast Study, Marketing Channels, Cost Structures, Distributors and Consumer Study

Section 11: Region-wise Forecast Analysis of Sales, Revenue, Growth Rate Till 2026

Section 12: Research Findings, Conclusion, Data Sources, Research Methodology, and Disclaimer

A complete qualitative and competitive assessment of Crispr And Crispr-Associated (Cas) Genes Market is conducted to offer valuable insights. This will enable the market aspirants in shaping their business plans and planning growth strategies. Primary and secondary research techniques like interviews, trade journals, surveys, and reputable paid database sources. A complete historical analysis from 2014-2019 and forecast analysis 2020-2026 with base year as 2019. Our competitive business landscape will help you to gain upper hand in competition.

About Us:

ReportsCheck.biz is one stop platform which caters to all your market research needs as well as custom and consulting services. We have an extensive database of research reports to meet the global, regional and country-level research requirements of our clients. We gather complete information about all products with the help of research methodology and verified data sources. We have an expert team to understand and map client requirements to provide precise research analysis. Our research solutions will help readers in aligning their business and analyzing business priorities.

Contact Us:

David Smith

Senior Manager Client Engagements

Phone No.: USA: +1 (831)-679-3317

Email ID: [emailprotected]

Read the rest here:
Comprehensive Crispr And Crispr-Associated (Cas) Genes Industry Outlook And Growth Trajectory Explained In This Latest Report - Jewish Life News

If you look back and reflect upon the areas of technology that werent conceived, or at least not commonplace, as we rolled into 2010 no iPads, Amazon Echo or Prime, smartwatches, Chromebooks, Uber, 3D printers, Instagram, Bitcoin then you will appreciate that a decade is a long time in tech.

Indeed, when you consider the pact of technological advancement and the theory that it is advancing exponentially, you can be sure that we will see changes just as dramatic, and perhaps more so, by the end of the 2020s.

Making tech predictions is, however, a good way to end up looking foolish. For instance, think about Microsoft CEO Steve Ballmer, who said in 2007, There is no chance that the iPhone is going to get any significant market share.

There are many ways to find out about your future and the direction you should take. But with technology, it can be explosive, with the changes feeling like they happen overnight. Below are some tech ideas which experts believe may be possible and commonplace by 2030:

Mass Use of Flying Jetpacks

Sick of the Memorial Day traffic in Cape Cod? Well, you can beat the rush by using your personal jet pack. YouTube is littered with inventors showing off their fabulous flying machines, but some serious engineers are getting behind the idea. In fact, the main issue is not the mechanics of flying, but the weight of fuel. Get that right, and the answer seems to lie with electricity, and you could be zipping around the Cape with no worries. Moreover, while we view the jetpack as a fun piece of science fiction, its use could have huge benefits for emergency services.

An AI Machine As Your Boss

It sounds like a nightmare dystopian future, but having a robot as your boss is not that far-fetched. A survey of tech and business experts in 2015 found that just under half (45.2%) predicted we would see an AI machine sitting on a board of directors of a corporation by 2025. The premise is that the machines can make wholly logical decisions, based on data analysis, that the human mind cant. While it may seem scary, experts maintain that AI is here to enhance our world, not replace us. We hope they are right.

Your CRISPR Children

CRISPR continuous regularly interspersed short palindromic repeats is really just a fancy name for gene editing. And, the advances in this area are set to cause a huge stir. We are perhaps not quite at the Jurassic Park level yet, but there will be able to do incredible things with it, not all of which will be considered ethical. For instance, it will be possible to edit the DNA sequences of your future children to give them desirable characteristics, just as it will be possible to create a pet lion the size of a house cat. You can see the sense in editing out a hereditary disease, but where do you draw the line?

Live Forever

Want to ensure that you are around for the Red Sox next World Series win should history repeat itself and the team receive another 86-year curse? Well, some very serious scientists believe the abolition of ageing is not that far off. Perhaps not widely available by 2030, although the theory will be firmed up by that time, but almost certainly by the middle of the century. Its a combination of DNA-editing and computers that hold the key, and youll probably get to house yourself within a sleek android body too.

Link:
These Far-Flung Tech Concepts Could Be a Reality by 2030 - Cape Cod Today

CAMBRIDGE, Mass., Feb. 03, 2020 (GLOBE NEWSWIRE) -- TCR2 TherapeuticsInc. (Nasdaq: TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, today announced that it will present a poster at the 2020 Keystone Symposia Conference on Emerging Cellular Therapies: Cancer and Beyond, taking place February 8-10, 2020 in Banff, Canada. The presentation will highlight allogeneic (off-the-shelf) T Cell Receptor Fusion Constructs (TRuC) T cells. In addition to utilizing TCR2s proprietary TRuC-T cell platform, the approach employs CRISPR/Cas9 endonucleases yielding fully functional TRuCs that lack alloreactivity and upregulate activation markers, secrete cytokines and kill tumor cells in an antigen-specific manner.

Presentation details are as follows:Title: Engineering Off-the-Shelf T Cell Receptor Fusion Construct (TRuC) T CellsPoster: 2002Session Title: Q2: Engineering the GenomeSession Date/Time: 7:30pm - 10:00pm M.S.T.

About TCR2 Therapeutics

TCR2Therapeutics Inc.is a clinical-stage immunotherapy company developing the next generation of novel Tcell therapies for patients suffering from cancer.TCR2sproprietary T cell receptor (TCR) Fusion Construct Tcells (TRuC-T cells) specifically recognize and kill cancer cells by harnessing signaling from the entire TCR, independent ofhuman leukocyte antigens (HLA). In preclinical studies, TRuC-T cells have demonstrated superior anti-tumor activity compared to chimeric antigen receptor T cells (CAR-T cells), while exhibiting lower levels of cytokine release. The Companys lead TRuC-T cell product candidate, TC-210, is currently being studied in a Phase 1/2 clinical trial to treat patients with mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. For more information about TCR2, please visitwww.tcr2.com.

Forward-looking Statements

This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include, but are not limited to, express or implied statements regarding the Companys TRuC-T cells, their potential characteristics, applications and clinical utility, and the potential therapeutic applications of the Companys TRuC-T cell platform.

The expressed or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical studies and in the availability and timing of data from ongoing clinical studies; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities, including review under accelerated approval processes; orphan drug designation eligibility; regulatory approvals to conduct trials or to market products; TCR2s ability to maintain sufficient manufacturing capabilities to support its research, development and commercialization efforts, whether TCR2's cash resources will be sufficient to fund TCR2's foreseeable and unforeseeable operating expenses and capital expenditure requirements; and other risks set forth under the caption "Risk Factors" in TCR2s most recent Annual Report on Form 10-K, most recent Quarterly Report on Form 10-Q and its other filings with theSecurities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although TCR2believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur.

Moreover, except as required by law, neither TCR2nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investor and Media Contact:

Carl MauchDirector, Investor Relations and Corporate Communications(617) 949-5667carl.mauch@tcr2.com

Read more from the original source:
TCR Therapeutics to Present an Allogeneic Construct at the Keystone Symposia Conference on Emerging Cellular Therapies: Cancer and Beyond - BioSpace

CRISPR technology is a simple yet powerful tool for editing genomes. It allows researchers to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. However, its promise also raises ethical concerns.

In popular usage, "CRISPR" (pronounced "crisper") is shorthand for "CRISPR-Cas9." CRISPRs are specialized stretches of DNA. The protein Cas9 (or "CRISPR-associated") is an enzyme that acts like a pair of molecular scissors, capable of cutting strands of DNA.

CRISPR technology was adapted from the natural defense mechanisms of bacteria and archaea (the domain of single-celled microorganisms). These organisms use CRISPR-derived RNA and various Cas proteins, including Cas9, to foil attacks by viruses and other foreign bodies. They do so primarily by chopping up and destroying the DNA of a foreign invader. When these components are transferred into other, more complex, organisms, it allows for the manipulation of genes, or "editing."

Until 2017, no one really knew what this process looked like. In a paper published Nov. 10, 2017, in the journal Nature Communications, a team of researchers led by Mikihiro Shibata of Kanazawa University and Hiroshi Nishimasu of the University of Tokyo showed what it looks like when a CRISPR is in action for the very first time. [A Breathtaking New GIF Shows CRISPR Chewing Up DNA]

CRISPRs: "CRISPR" stands for "clusters of regularly interspaced short palindromic repeats." It is a specialized region of DNA with two distinct characteristics: the presence of nucleotide repeats and spacers. Repeated sequences of nucleotides the building blocks of DNA are distributed throughout a CRISPR region. Spacers are bits of DNA that are interspersed among these repeated sequences.

In the case of bacteria, the spacers are taken from viruses that previously attacked the organism. They serve as a bank of memories, which enables bacteria to recognize the viruses and fight off future attacks.

This was first demonstrated experimentally by Rodolphe Barrangou and a team of researchers at Danisco, a food ingredients company. In a 2007 paper published in the journal Science, the researchers used Streptococcus thermophilus bacteria, which are commonly found in yogurt and other dairy cultures, as their model. They observed that after a virus attack, new spacers were incorporated into the CRISPR region. Moreover, the DNA sequence of these spacers was identical to parts of the virus genome. They also manipulated the spacers by taking them out or putting in new viral DNA sequences. In this way, they were able to alter the bacteria's resistance to an attack by a specific virus. Thus, the researchers confirmed that CRISPRs play a role in regulating bacterial immunity.

CRISPR RNA (crRNA): Once a spacer is incorporated and the virus attacks again, a portion of the CRISPR is transcribed and processed into CRISPR RNA, or "crRNA." The nucleotide sequence of the CRISPR acts as a template to produce a complementary sequence of single-stranded RNA. Each crRNA consists of a nucleotide repeat and a spacer portion, according to a 2014 review by Jennifer Doudna and Emmanuelle Charpentier, published in the journal Science.

Cas9: The Cas9 protein is an enzyme that cuts foreign DNA.

The protein typically binds to two RNA molecules: crRNA and another called tracrRNA (or "trans-activating crRNA"). The two then guide Cas9 to the target site where it will make its cut. This expanse of DNA is complementary to a 20-nucleotide stretch of the crRNA.

Using two separate regions, or "domains" on its structure, Cas9 cuts both strands of the DNA double helix, making what is known as a "double-stranded break," according to the 2014 Science article.

There is a built-in safety mechanism, which ensures that Cas9 doesn't just cut anywhere in a genome. Short DNA sequences known as PAMs ("protospacer adjacent motifs") serve as tags and sit adjacent to the target DNA sequence. If the Cas9 complex doesn't see a PAM next to its target DNA sequence, it won't cut. This is one possible reason that Cas9 doesn't ever attack the CRISPR region in bacteria, according to a 2014 review published in Nature Biotechnology.

The genomes of various organisms encode a series of messages and instructions within their DNA sequences. Genome editing involves changing those sequences, thereby changing the messages. This can be done by inserting a cut or break in the DNA and tricking a cell's natural DNA repair mechanisms into introducing the changes one wants. CRISPR-Cas9 provides a means to do so.

In 2012, two pivotal research papers were published in the journals Science and PNAS, which helped transform bacterial CRISPR-Cas9 into a simple, programmable genome-editing tool.

The studies, conducted by separate groups, concluded that Cas9 could be directed to cut any region of DNA. This could be done by simply changing the nucleotide sequence of crRNA, which binds to a complementary DNA target. In the 2012 Science article, Martin Jinek and colleagues further simplified the system by fusing crRNA and tracrRNA to create a single "guide RNA." Thus, genome editing requires only two components: a guide RNA and the Cas9 protein.

"Operationally, you design a stretch of 20 [nucleotide] base pairs that match a gene that you want to edit," said George Church, a professor of genetics at Harvard Medical School. An RNA molecule complementary to those 20 base pairs is constructed. Church emphasized the importance of making sure that the nucleotide sequence is found only in the target gene and nowhere else in the genome. "Then the RNA plus the protein [Cas9] will cut like a pair of scissors the DNA at that site, and ideally nowhere else," he explained.

Once the DNA is cut, the cell's natural repair mechanisms kick in and work to introduce mutations or other changes to the genome. There are two ways this can happen. According to the Huntington's Outreach Project at Stanford (University), one repair method involves gluing the two cuts back together. This method, known as "non-homologous end joining," tends to introduce errors. Nucleotides are accidentally inserted or deleted, resulting in mutations, which could disrupt a gene. In the second method, the break is fixed by filling in the gap with a sequence of nucleotides. In order to do so, the cell uses a short strand of DNA as a template. Scientists can supply the DNA template of their choosing, thereby writing-in any gene they want, or correcting a mutation.

CRISPR-Cas9 has become popular in recent years. Church notes that the technology is easy to use and is about four times more efficient than the previous best genome-editing tool (called TALENS).

In 2013, the first reports of using CRISPR-Cas9 to edit human cells in an experimental setting were published by researchers from the laboratories of Church and Feng Zhang of the Broad Institute of the Massachusetts Institute of Technology and Harvard. Studies using in vitro (laboratory) and animal models of human disease have demonstrated that the technology can be effective in correcting genetic defects. Examples of such diseases include cystic fibrosis, cataracts and Fanconi anemia, according to a 2016 review article published in the journal Nature Biotechnology. These studies pave the way for therapeutic applications in humans.

"I think the public perception of CRISPR is very focused on the idea of using gene editing clinically to cure disease," said Neville Sanjana of the New York Genome Center and an assistant professor of biology, neuroscience and physiology at New York University. "This is no doubt an exciting possibility, but this is only one small piece."

CRISPR technology has also been applied in the food and agricultural industries to engineer probiotic cultures and to vaccinate industrial cultures (for yogurt, for example) against viruses. It is also being used in crops to improve yield, drought tolerance and nutritional properties.

One other potential application is to create gene drives. These are genetic systems, which increase the chances of a particular trait passing on from parent to offspring. Eventually, over the course of generations, the trait spreads through entire populations, according to the Wyss Institute. Gene drives can aid in controlling the spread of diseases such as malaria by enhancing sterility among the disease vector female Anopheles gambiae mosquitoes according to the 2016 Nature Biotechnology article. In addition, gene drives could also be used to eradicate invasive species and reverse pesticide and herbicide resistance, according to a 2014 article by Kenneth Oye and colleagues, published in the journal Science.

However, CRISPR-Cas9 is not without its drawbacks.

"I think the biggest limitation of CRISPR is it is not a hundred percent efficient," Church told Live Science. Moreover, the genome-editing efficiencies can vary. According to the 2014 Science article by Doudna and Charpentier, in a study conducted in rice, gene editing occurred in nearly 50 percent of the cells that received the Cas9-RNA complex. Whereas, other analyses have shown that depending on the target, editing efficiencies can reach as high as 80 percent or more.

There is also the phenomenon of "off-target effects," where DNA is cut at sites other than the intended target. This can lead to the introduction of unintended mutations. Furthermore, Church noted that even when the system cuts on target, there is a chance of not getting a precise edit. He called this "genome vandalism."

The many potential applications of CRISPR technology raise questions about the ethical merits and consequences of tampering with genomes.

In the 2014 Science article, Oye and colleagues point to the potential ecological impact of using gene drives. An introduced trait could spread beyond the target population to other organisms through crossbreeding. Gene drives could also reduce the genetic diversity of the target population.

Making genetic modifications to human embryos and reproductive cells such as sperm and eggs is known as germline editing. Since changes to these cells can be passed on to subsequent generations, using CRISPR technology to make germline edits has raised a number of ethical concerns.

Variable efficacy, off-target effects and imprecise edits all pose safety risks. In addition, there is much that is still unknown to the scientific community. In a 2015 article published in Science, David Baltimore and a group of scientists, ethicists and legal experts note that germline editing raises the possibility of unintended consequences for future generations "because there are limits to our knowledge of human genetics, gene-environment interactions, and the pathways of disease (including the interplay between one disease and other conditions or diseases in the same patient)."

Other ethical concerns are more nuanced. Should we make changes that could fundamentally affect future generations without having their consent? What if the use of germline editing veers from being a therapeutic tool to an enhancement tool for various human characteristics?

To address these concerns, the National Academies of Sciences, Engineering and Medicine put together a comprehensive report with guidelines and recommendations for genome editing.

Although the National Academies urge caution in pursuing germline editing, they emphasize "caution does not mean prohibition." They recommend that germline editing be done only on genes that lead to serious diseases and only when there are no other reasonable treatment alternatives. Among other criteria, they stress the need to have data on the health risks and benefits and the need for continuous oversight during clinical trials. They also recommend following up on families for multiple generations.

There have been many recent research projects based around CRISPR. "The pace of basic research discoveries has exploded, thanks to CRISPR," said biochemist and CRISPR expert Sam Sternberg, the group leader of technology development at Berkeley, California-based Caribou Biosciences Inc., which is developing CRISPR-based solutions for medicine, agriculture, and biological research.

Here are some of the most recent findings:

Additional reporting by Alina Bradford, Live Science contributor.

Additional resources

View post:
What Is CRISPR? | Live Science

CRISPR Technology Market to 2027 - Global Analysis and Forecasts By Product and Services (Enzymes, Kits, Services and Others), Application (Genetic Engineering, Cell Line Engineering and Others) End User (Biotechnology & Pharmaceutical Companies, Contract Research Organizations (CROs), and Academic & Government Research Institutes); and Geography

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology is a simple but powerful tool for genome editing. This tool enables life science researchers to easily edit DNA sequences and modify gene function.

It has many potential applications include correcting genetic defects, treating and preventing the spread of diseases and improving crops. By delivering the CRISPR enzyme Cas9 nuclease coupled with synthetic guide RNA (gRNA) into a cell, the cell's genome can be cut at a desired location, that allows existing genes to be removed or add new ones.

Increasing usage of CRISPR systems in microbiology, growing government and private investments on research and development of genome editing, rising prevalence of genetic disorders, and increases application of CRISPR/Cas9 technology to improve crop production drives the global CRISPR technology market. However, ethical issues associated with CRISPR and lack of skilled personnel restrain the global CRISPR technology market over the forecast period.

Download Samle PDF Of Report@www.theinsightpartners.com/sample/tech-10186

Some of the key players operating in the CRISPR Technology market include :-

Thermo Fisher Scientific Inc., Merck KGaA, Horizon Discovery Group plc, Cellecta, Inc, GeneCopoeia, Inc., New England Biolabs, OriGene Technologies, Inc., GenScript, Integrated DNA Technologies, Inc. and Agilent Technologies, Inc.

The report also includes the profiles of key CRISPR Technology companies along with their SWOT analysis and market strategies.

In addition, the report focuses on leading industry players with information such as company profiles, components and services offered, financial information of last 3 years, key development in past five years.

The "Global CRISPR Technology Market Analysis to 2027" is a specialized and in-depth study of the medical device industry with a focus on the global market trend. The report aims to provide an overview of global market with detailed market segmentation by product and services, application, end user and geography.

The global CRISPR Technology market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading CRISPR Technology market players and offers key trends and opportunities in the market.

The global CRISPR technology market is segmented on the basis of product and services, application, end user. Based product and services, the market is segmented as, enzymes, kits, services and others.

The CRISPR technology market is categorized based on application into, genetic engineering, cell line engineering and others. Based on end user, the CRISPR Technology market is classified into biotechnology & pharmaceutical companies, contract research organizations (CROS), and academic & government research institutes.

The report provides a detailed overview of the industry including both qualitative and quantitative information. It provides overview and forecast of the global CRISPR Technology market based product and services, application, end user.

It also provides market size and forecast till 2027 for overall market with respect to five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America. The CRISPR Technology Market by each region is later sub-segmented by respective countries and segments.

The report covers analysis and forecast of 13 countries globally along with current trend and opportunities prevailing in the region.

North America held over major share in the CRISPR Technology market in 2017 owing to significant research carried out in order to develop novel therapeutics for disease targeting and high adoption of genome editing technique for germline modifications. North America is expected to collectively contribute towards the growth of CRISPR Technology market owing to the presence of major market players and also the development of technologically advanced products of CRISPR technology is expected to influence the CRISPR technology market growth.

The Asia-Pacific region is expected to exhibit highest CAGR during the forecast period due to many applications in developing economies of the region for animal disease and human disease treatment. Also, the rapid economic growth in this region coupled with diversified population and large patient pool, drives CRISPR Technology market in this region.

The report analyzes factors affecting CRISPR Technology market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the CRISPR Technology market in these regions.

Access Full Report@www.theinsightpartners.com/buy/TIPtech-10186

This email address is being protected from spambots. You need JavaScript enabled to view it.

See more here:
CRISPR Technology Market analysis by growth, segmentation, performance, competitive strategies and forecast to 2027 - WhaTech Technology and Markets...

Mammoth Biosciences cofounders Janice Chen, Lucas Harrington and Trevor Martin.

Mammoth Biosciences, a company that uses gene-editing technology Crispr for disease testing, said Thursday that it had raised $45 million in Series B funding to expand into treatments. The round, led by Decheng Capital and including new investor Verily, brings total funding to over $70 million.

The South San Francisco-based company, founded in 2017 by Forbes Under 30 honorees Trevor Martin, Janice Chen, Lucas Harrington and Crispr pioneer Jennifer Doudna, uses Crispr as a genetic search engine to find disease markers and alert researchers of their presence. Theyve already partnered with others, such as gene-editing company Horizon Discovery and a UC San Francisco researcher who is creating a rapid diagnostic test to identify people infected with the new coronavirus.

The company has been one of the most prolific innovators in the overall Crispr ecosystem, says Ursheet Parikh, an investor at the Mayfield Fund, which also participated in the round.

The new capital will allow Mammoth to expand into more traditional gene editing, which can be used to treat diseases. The company also plans to double in size, Martin says. Mammoth has already moved into new lab space on the South San Francisco campus of Verily, Alphabets life sciences company.

Crispr gene editing emerged in the 2010s as a tool that could quickly and precisely snip, repair or insert genes into DNA, giving rise to companies including eGenesis, Caribou Biosciences and Sherlock Biosciences. Most biotech companies in the gene-editing space use the Crispr system with Cas9, a large protein that can cut DNA. Mammoth focuses on a different one: Cas14. Martin refers to this protein as nano-Cas, because its smaller and more precise than the popular Cas9 protein. Its more of a scalpel than a sledgehammer, he says. In a diagnostic test, the Cas protein is programmed to find a specific target. Once it finds this target, it breaks apart a reporter molecule, which can then change the color of the solution, indicating a positive or negative test result. Cas14 is particularly useful in diagnostics, Martin says, because of its size and its ability to quickly generate a signal once it finds DNA evidence of disease.

The technology has big implications for diagnostics, Martin says. One of Mammoths current partnerships is with UCSF researcher Charles Chiu, who also sits on Mammoths scientific advisory board, to create a rapid diagnostic test for the new coronavirus that has sickened more than 6,100 people globally and killed 132.

Right now, suspected coronavirus samples are shipped to the Centers for Disease Control and Prevention, where it can take six or more hours for the test to complete. The new test will work by taking a sample from a nasal swab, putting it into a tube with the Crispr-Cas system and other chemicals, and then dipping in a color-changing strip of paper to determine whether the test result is positive or negative. The whole thing should take from one to two hours, Chiu says, and be done in a doctors clinic or an emergency room. His lab was already working on a similar diagnostic test for Lyme disease, and it was able to adapt the test quickly to the new coronavirus. Chiu says the test could be ready in a matter of weeks; the only thing holding it back is a lack of human samples with which to test the diagnostic accuracy. Chiu credits Mammoths platform for helping them create a better, faster test. There are very few if any technologies that you could use that would have the same speed, turnaround and accuracy, he says.

Originally posted here:
Mammoth Biosciences Raises $45 Million For Crispr DiagnosticsAnd Its Tech Is Already Being Used Against Coronavirus - Forbes

Researchers at the University of North Carolina at Chapel Hill say they can detect autism spectrum disorder before it manifests in some young children, and theyre even developing treatments for some of the conditions that go hand-in-hand with autism.

Professors Mark Zylka and Joe Piven work among more than two dozen scientists at UNC focused on autism spectrum disorder, and the National Institutes of Health have given the two more than $15 million combined in the last year alone.

Autism is a developmental disorder that affects communication and behavior, with symptoms that can include repetitive behavior and difficulty interacting with other people.

Piven has been able to detect autism in children as young as six months. And Zylka has focused on treating a syndrome closely linked to autism with gene editing, research he says could open the door to a much broader slate of treatments.

There's been a real revolution in the past 10 years in terms of our understanding of the genetic basis for autism, says Zylka, who studies cell biology and physiology. This revolution has really been sparked by the rapidly reducing cost of sequencing human genomes.

Just this month, the largest genetic study of autism to date found more than 100 genes linked to the disorder. Those kinds of breakthroughs are stepping stones toward a better understanding of autism spectrum disorder, the researchers say.

In addition to genetics, environmental factors including maternal health and prenatal exposure to air pollution play a role in the development of autism. The disorder is diagnosed in one in every 59 children in the United States, according to the Centers for Disease Control and Prevention. Two decades ago, the rate of diagnosis was just 1 in 150 children.

Piven, who studies psychiatry and pediatrics, says its hard to tell why the prevalence is increasing, though better recognition and widening criteria likely play a large role. But the increased prevalence has also generated enthusiasm for public and private funding, he says.

Earlier Is Better

With that funding including a $9.5 million grant from the NIH last year Piven and his team have been looking at the brains of kids six to 12 months old.

He uses MRI brain-imaging to predict whether a child will develop autism, well before children turn two or three and start showing symptoms. The kids hes studying have older siblings diagnosed with autism, so they run a much higher risk than the average child of developing it themselves about a 20% likelihood.

But babies who later develop autism spectrum disorder don't look like they have autism in the first year of life, he says. That's really quite amazing. And that window gives us an opportunity to think about early detection.

While the infants dont exhibit symptoms of autism, their brains look different from children who dont develop the disorder, Piven says, including differences in surface anatomy, surface area, convolutions on the surface.

Those variations have allowed him to correctly identify eight out of 10 kids who would go on to develop autism in previous studies. He says that predictive tool could allow researchers to develop early interventions.

Earlier is better, he says. As a rule of thumb in medicine, we treat things before they happen. ... We are interested in high blood pressure because it leads to stroke, so we treat high blood pressure. And that's a well-worn path.

The interventions themselves are still an open question, he says, because researchers haven't been able to identify these children in infancy before.

Turning Genes On And Off

One potential treatment, though, is gene editing.

Thats where Zylka comes in. His research, also funded by the NIH, involves mice instead of children for now. Hes trying to treat Angelman syndrome, a rare neurodevelopmental disorder often placed on the autism spectrum.

These are children that are largely non-verbal, Zylka says. They have motor problems. Its severely disabling.

People with Angelman syndrome have a mutation in the maternal UBE3A gene. Normally, any given gene passed down from one parent doesnt have to function perfectly because theres a backup the other parents gene.

But the paternal UBE3A gene is largely inactive, or silenced. Thats fine for most of us, but it means theres no backup for a child born with a missing or defective maternal gene.

The paternal gene is functional, but turned off, Zylka says. Using these new genome editing technologies like CRISPR-Cas, we're going in and trying to turn on that dad's copy of the gene.

CRISPR has gained international acclaim for its promise in treating disease, but it has also generated controversy. A Chinese researcher who said he had illegally created the world's first gene-edited babies was sentenced to prison last year.

But Zylka says his team has figured out ways to harness the tools power to treat Angelman syndrome without creating mutations that could be passed onto future generations.

So far, hes been able to treat symptoms in mice, though not eradicate them and he says the treatment has to be administered early in life to work properly. Eventually, he hopes to use CRISPR-Cas9 to edit the UBE3A genes of prenatal infants or newborns.

Zylka says hes not finding a cure per se an idea that many people with autism and advocates oppose but trying to treat potentially devastating symptoms: Angelman syndrome can cause epilepsy and severe speech impairment.

If you have a baby and at birth, they have some problem that surgery can correct, people are not going to neglect the surgery to fix the baby, he says. With genetics, we can actually pick these mutations up early, so ... gene editing approaches could be used to treat early.

He says this work could open the door to treating autism more broadly.

CRISPR-Cas technology can be used to turn genes off or turn genes on, Zylka says. Since many cases of autism are due to loss of one copy, you still have a second copy that is functional. And so you could use an editing approach to turn on the functional copy to a higher level.

Both Piven and Zylka say public funding from the NIH is a mainstay for their work. And theyre optimistic about the future of autism research, especially as more comes to light about the causes of autism syndrome disorder and the variations within that diagnosis.

While we call this autism ... these really aren't [all] the same condition, says Piven. We just have these crude behavioral criteria. So I think we will pick away at the whole and start being very successful with some that have these more simple mechanisms. And others that are more complicated, we'll have to tackle in other ways.

Zylka says subtyping disorders might even let researchers come up with personalized treatment one day. But, he adds, were not there yet.

Find more information about recruitment for this study here.

Francesca Parisproduced and edited this interview for broadcast withKathleen McKenna. Paris also adapted it for the web.

Go here to read the rest:
On The Frontlines Of Autism Research: North Carolina Professors Study Early Detection, Treatment - Here And Now

As infectious disease specialists and epidemiologists race to contain the outbreak of the novel coronavirus centered on Wuhan, China, theyre getting backup thats been possible only since the explosion in genetic technologies: a deep-dive into the genome of the virus known as 2019-nCoV.

Analyses of the viral genome are already providing clues to the origins of the outbreak and even possible ways to treat the infection, a need that is becoming more urgent by the day: Early on Saturday in China, health officials reported 15 new fatalities in a single day, bringing the death toll to 41. There are now nearly 1,100 confirmed cases there.

Reading the genome (which is made of RNA, not DNA) also allows researchers to monitor how 2019-nCoV is changing and provides a roadmap for developing a diagnostic test and a vaccine.

advertisement

The genetics can tell us the true timing of the first cases and whether they occurred earlier than officials realized, said molecular biologist Kristian Andersen of Scripps Research, an expert on viral genomes. It can also tell us how the outbreak started from a single event of a virus jumping from an infected animal to a person or from a lot of animals being infected. And the genetics can tell us whats sustaining the outbreak new introductions from animals or human-to-human transmission.

Scientists in China sequenced the viruss genome and made it available on Jan. 10, just a month after the Dec. 8 report of the first case of pneumonia from an unknown virus in Wuhan. In contrast, after the SARS outbreak began in late 2002, it took scientists much longer to sequence that coronavirus. It peaked in February 2003 and the complete genome of 29,727 nucleotides wasnt sequenced until that April.

Since the sequencing of the first 2019-nCoV sample, from an early patient, scientists have completed nearly two dozen more, said Andrew Rambaut of the University of Edinburgh, an expert on viral evolution. That pace is unprecedented and completely unbelievable, said Andersen, who worked on sequencing the Ebola genome during the 2014 outbreak. Its just insane.

The genome of the Wuhan virus is 29,903 bases long, one of many clues that have led scientists to believe it is very similar to SARS.

By comparing the two dozen genomes, scientists can address the when did this start question. The 24 available samples, including from Thailand and Shenzhen as well as Wuhan, show very limited genetic variation, Rambaut concluded on an online discussion forum where virologists have been sharing data and analyses. This is indicative of a relatively recent common ancestor for all these viruses.

Given whats known about the pace at which viral genomes mutate, if nCoV had been circulating in humans since significantly before the first case was reported on Dec. 8, the 24 genomes would differ more. Applying ballpark rates of viral evolution, Rambaut estimates that the Adam (or Eve) virus from which all others are descended first appeared no earlier than Oct. 30, 2019, and no later than Nov. 29.

The progenitor virus itself was almost certainly one that circulates harmlessly in bats (as SARS does) but has an intermediate reservoir in one or more animals that come into contact with people, Andersen said. Presumably, that reservoir is one of the species of animals at the Wuhan market thought to be ground zero for the outbreak. The ancestor of 2019-nCoV existed in that species for some unknown time, never infecting people, until by chance a single virus acquired a mutation that made it capable of jumping into and infecting humans.

The genome sequences suggest that was a one-time-only jump. The genomes [from the 24 samples] are very uniform, Andersen said. If there had been multiple introductions, including from many different animals, there would be more genomic diversity. This was a single introduction.

That means that whats sustaining the spread is human-to-human transmission (suggesting that closing Wuhans animal market is very much an after-the-horse-has-fled-the-barn reaction).

Unfortunately, genetic analysis cant identify what animal species the coronavirus jumped from into humans. But an analysis by a team from the Wuhan Institute of Virology, posted to the preprint server bioRxiv, determined that the genome of this coronavirus (the seventh known to infect humans) is 96% identical to that of a bat coronavirus, suggesting that species is the original source. (Writing in the New England Journal of Medicine on Friday, another team of scientists in China reported that the new coronavirus is 86.9% identical to the bat SARS-like coronavirus.)

Virologists differ on whether its possible to read out viral properties from just the genome sequence, such as whether the microbe is spread by coughing, sneezing, touching,or merely breathing. But the analysis by the Wuhan Institute team found that it enters human cells using the same doorway that SARS did. Called angiotensin converting enzyme 2 (ACE2), the door is a receptor to which a spike protein on the viruss surface first attaches and then enables the virus to fuse with the host cell.

If ACE2 is druggable, blocking it could conceivably treat 2019-nCoV. It should be expected and worth to test if ACE2 targeting drugs can be used for nCoV-2019 patients, the scientists wrote.

The genome sequences have more to give. They will be crucially important for development of diagnostics [and] vaccines, said biologist Richard Ebright of Rutgers University.

For instance, the genome-editing technology CRISPR is the basis for Cambridge, Mass.-based startup Sherlock Biosciences diagnostics, which promise to slash how long it takes to make a definitive identification. In the U.S, thats now done only by sending samples to the Centers for Disease Control and Prevention, which uses a technology invented in the 1980s, polymerase chain reaction or PCR, to identify the presence of coronavirus.

Our vision is that our [CRISPR-based] SHERLOCK and INSPECTR platforms are tailor-made for outbreaks like coronavirus, said Sherlock CEO Rahul Dhanda, who declined to discuss specific plans related to coronavirus.

And as scientists keep adding 2019-nCoV genome sequences to their collection, they could get an early glimpse of whether the virus is mutating in a way that could make it more dangerous or more transmissible. You need continuous sequencing, Andersen said.

Correction: This story has been corrected to make clear that the coronavirus genome is made of RNA, not DNA.

Continued here:
DNA sleuths read the coronavirus genome, tracing its origins - STAT

The non-profit science NGO Genetic Literacy Project has released its latest educational initiative, the Global Gene Editing Regulation Tracker and Index.

With the worldwide war on GMOs essentially lost by environmental lawyers, they still continue to hold back Europe but developing nations have seen through the false promises of western activists who have no solutions to poverty and food insecurity, only fear of the future. They are becoming hopeful about the future.

Thanks to CRISPR-Cas9 gene editing, non-chemical solutions to life-impacting developing nation problems such as malaria (dengue, yellow fever) mosquitoes can be developed, and governments will be scrambling to adapt a regulatory structure that meets the 21st century.

In the past, anti-science NGOs were able to successfully frame GMOs as too modern and terrifying. They had to ignore the existence of Mutagenesis, chemical and radiation baths used to create new strains of food and plant products in the lab, because those biotechnology results are considered part of an organic scheme. GMOs were different, they insisted.

So now they have to scramble to claim GMOs are different from mutagenesis and yet the same as CRISPR, even though they all share little in common beyond being ways to improve on nature.

So much information and disinformation can be confusing for the public. The new Genetic Literacy Project program summarizes gene editing regulations in each country's agriculture, medicine and gene efforts, along with what products and therapies are in development.

Most importantly for real progress, it also details the efforts by anti-science NGOs to block progress.

Read the rest here:
Genetic Literacy Project Releases Global Gene Editing Regulation Tracker and Index - Science 2.0

The recent performance of Aphria (NYSE:APHA) stock in the market spoke loud and clear to investors as APHA saw more than 5.58M shares in trading volumes in the last trading session, way higher than the average trading volume of 5.58M shares by far recorded in the movement of Aphria (APHA). At the time the stock opened at the value of $5.17, making it a high for the given period, the value of the stock dropped by -7.97%. After the decrease, APHA touched a low price of $4.85, calling it a day with a closing price of $5.27, which means that the price of APHA went 4.85 below the opening price on the mentioned day.

Other indicators are hinting that the stock could reach an outstanding figure in the market share, which is currently set at 252.56M in the public float and 1.23B US dollars in market capitalization.

When it comes to the technical analysis of APHA stock, there are more than several important indicators on the companys success in the market, one of those being the Relative Strength Indicator (RSI), which can show, just as Stochastic measures, what is going on with the value of the stock beneath the data. This value may also indicate that the stock will go sideways rather than up or down, also indicating that the price could stay where it is for quite some time. When it comes to Stochastic reading, APHA stock are showing 52.63% in results, indicating that the stock is neither overbought or oversold at the moment, providing it with a neutral within Stochastic reading as well. Additionally, APHA with the present state of 200 MA appear to be indicating bearish trends within the movement of the stock in the market. While other metrics within the technical analysis are due to provide an outline into the value of APHA, the general sentiment in the market is inclined toward negative trends.

With the previous 100-day trading volume average of 931609 shares, CRISPR Therapeutics AG (CRSP) recorded a trading volume of 996420 shares, as the stock started the trading session at the value of $54.75, in the end touching the price of $53.59 after dropping by -2.12%.

CRSP stock seem to be going ahead the lowest price in the last 52 weeks with the latest change of 82.65%.Then price of CRSP also went backward in oppose to its average movements recorded in the previous 20 days. The price volatility of CRSP stock during the period of the last months recorded 4.56%, whilst it changed for the week, now showing 4.30% of volatility in the last seven days. The trading distance for this period is set at -10.90% and is presently away from its moving average by -15.44% in the last 50 days. During the period of the last 5 days, CRSP stock lost around -8.13% of its value, now recording a sink by 9.89% reaching an average $48.84 in the period of the last 200 days.During the period of the last 12 months, CRISPR Therapeutics AG (CRSP) dropped by -12.01%.

According to the Barcharts scale, the companys consensus rating fall to 4.27 from 4.60, showing an overall improvement during the course of a single month. Based on the latest results, analysts are suggesting that the target price for CRSP stock should be $53.59 per share in the course of the next 12 months. To achieve the target price as suggested by analysts, CRSP should have a spike by 0% in oppose to its present value in the market. Additionally, the current price showcases a discount of 48.47% when compared to the high consensus price target predicted by analysts.

CRSP shares recorded a trading volume of 834200 shares, compared to the volume of 1.22M shares before the last close, presented as its trading average. With the approaching 4.30% during the last seven days, the volatility of CRSP stock remained at 4.56%. During the last trading session, the lost value that CRSP stock recorded was set at the price of $53.59, while the lowest value in the last 52 weeks was set at $29.34. The recovery of the stock in the market has notably added 82.65% of gains since its low value, also recording -20.29% in the period of the last 1 month.

Go here to see the original:
Why Investors Rotating Towards Aphria (APHA), CRISPR Therapeutics AG (CRSP) - US Post News

CRISPR, the breakthrough method for editing genes, has the potential to improve our lives. But one of its inventors warns us scientists may be tempted to change life itself in ways we wont like.

Jennifer Doudna, biochemist who helped invent CRISPR technology. Professor of chemistry, biochemistry and molecular biology at the University of California, Berkeley. (@doudna_lab)

Alta Charo, member of the WHO's advisory committee on developing global standards for governance and over-sight of human genome editing. 2019-2020 Berggruen fellow at theCenter for Advanced Study in Behavioral Sciencesat Stanford University. (@CASBSStanford)

Science Magazine: "Editorial: CRISPR's unwanted anniversary" "There are key moments in the history of every disruptive technology that can make or break its public perception and acceptance. For CRISPR-based genome editing, such a moment occurred 1 year agoan unsettling push into an era that will test how society decides to use this revolutionary technology.

"In November 2018, at the Second International Summit on Human Genome Editing in Hong Kong, scientist He Jiankui announced that he had broken the basic medical mantra of 'do no harm' by using CRISPR-Cas9 to edit the genomes of two human embryos in the hope of protecting the twin girls from HIV.

"His risky and medically unnecessary work stunned the world and defied prior calls by my colleagues and me, and by the U.S. National Academies of Sciences and of Medicine, for an effective moratorium on human germline editing. It was a shocking reminder of the scientific and ethical challenges raised by this powerful technology.

"Once the details of He's work were revealed, it became clear that although human embryo editing is relatively easy to achieve, it is difficult to do well and with responsibility for lifelong health outcomes."

MIT Technology Review: "One of CRISPRs inventors has called for controls on gene-editing technology" "Regulators need to pay more attention to controlling CRISPR, the revolutionary gene-editing tool, says Jennifer Doudna.One year on: Doudna, a University of California biochemist who helped invent CRISPR technology in 2012, wrote an editorial in Science yesterday titled CRISPRs unwanted anniversary.

"The anniversary is that of the announcement by a Chinese scientist, He Jiankui, that he had created gene-edited twin girls. That was a medical felony as far as Doudna is concerned, an unnecessary experiment that violated the doctors rule to avoid causing harm and ignored calls not to proceed.

"A moratorium? Forget about it. So how do we stop this from happening again? Since the 'CRISPR babies' debacle, scientists have talked about self-regulation. One idea was a moratorium: a self-imposed ban of a few years before anyone tries using the technology on the human germline again. (The germline refers to embryos, sperm, and eggsanything that, if you edit it, will cause changes that pass down through the generations.) But that's not going to cut it, says Doudna."

The New York Times: "Jennifer Doudna, a Pioneer Who Helped Simplify Genome Editing" "As a child in Hilo, one of the less touristy parts of Hawaii, Jennifer A. Doudna felt out of place. She had blond hair and blue eyes, and she was taller than the other kids, who were mostly of Polynesian and Asian descent.

'I think to them I looked like a freak,' she recently recalled. 'And I felt like a freak.'Her isolation contributed to a kind of bookishness that propelled her toward science. Her upbringing 'toughened her up,' said her husband, Jamie Cate. 'She can handle a lot of pressure.' These days, that talent is being put to the test.

"Three years ago, Dr. Doudna, a biochemist at the University of California, Berkeley, helped make one of the most monumental discoveries in biology: a relatively easy way to alter any organisms DNA, just as a computer user can edit a word in a document.

"The discovery has turned Dr. Doudna (the first syllable rhymes with loud) into a celebrity of sorts, the recipient of numerous accolades and prizes. The so-called Crispr-Cas9 genome editing technique is already widely used in laboratory studies, and scientists hope it may one day help rewrite flawed genes in people, opening tremendous new possibilities for treating, even curing, diseases."

The New York Times: "Chinese Scientist Who Genetically Edited Babies Gets 3 Years in Prison" "A court in China on Monday sentenced He Jiankui, the researcher who shocked the global scientific community when he claimed that he had created the worlds first genetically edited babies, to three years in prison for carrying out 'illegal medical practices.'

"In a surprise announcement from a trial that was closed to the public, the court in the southern city of Shenzhen found Dr. He guilty of forging approval documents from ethics review boards to recruit couples in which the man had H.I.V. and the woman did not, Xinhua, Chinas official news agency, reported. Dr. He had said he was trying to prevent H.I.V. infections in newborns, but the state media on Monday said he deceived the subjects and the medical authorities alike.

"Dr. He, 35, sent the scientific world into an uproar last year when he announced at a conference in Hong Kong that he had created the worlds first genetically edited babies twin girls. On Monday, Chinas state media said his work had resulted in a third genetically edited baby, who had been previously undisclosed."

Excerpt from:
CRISPR Has The Potential To Improve Lives. But At What Cost? - WBUR

New findings using CRISPR have shown that the IL-4 and IL-13 proteins can protect the body against inflammation from autoimmune diseases.

Proteins that play a role in allergies and parasitic infection can also stop the immune system from attacking the body and causing inflamed joints. The researchers suggest their findings will give rise to new drugs for autoimmune diseases such as rheumatoid arthritis.

these proteins prevent neutrophils from migrating into the inflamed join

The investigation, from the Karolinska Institutet, Sweden, revealed that the IL-4 and IL-13 proteins can aid in preventing autoimmune attacks.

These proteins are secreted by immune cells in the presence or allergens or parasitic infections. This then influences the behaviour of neutrophils, a specific type of immune cell. Neutrophils are commonly found in the actively inflamed joints of patients with rheumatoid arthritis and are particularly virulent against tissue as they can secrete non-specific tissue irritants.

Previous research has shown that IL-4 and IL-13 can affect arthritis in experimental models, but exactly how they do so has remained unknown.

The results of this latest study show that these proteins prevent neutrophils from migrating into the inflamed joint. Using CRISPR to modify selected immune-cell genes to understand how they affect cell behaviour, the researchers found that the presence of IL-4 or IL-13 also stimulates an increase in neutrophil surface receptors which have an inhibiting effect on joint inflammation.

We will continue to study these mechanisms and hope that our work can contribute to the development of treatments for rheumatoid arthritis, said principal investigatorDr Fredrik Wermeling, assistant professor at theDepartment of Medicine, Karolinska Institutet.

I have high hopes that the experimental use of CRISPR will be hugely important to our understanding of how immune-cell behaviour is regulated and that this can guide us in the development of new efficacious drugs, concluded Wermeling.

The findings were published in PNAS.

Link:
Researchers use CRISPR to identify proteins that prevent inflammation - Drug Target Review

Issued on: 24/01/2020 - 17:12Modified: 24/01/2020 - 17:13

It's called CRISPR-Cas 9 and while the name may not sound impressive, don't be mistaken:this gene-editing technology is set to change our world in many unpredictable ways. We take a closer look in this edition of Tech 24.

It's often referred to as "DNA scissors". CRISPR-Cas 9 is a powerful tool that scientists can use to edit DNA and modify gene functions. It was created byresearchersJennifer Doudnaand Emmanuelle Charpentier in 2012 and it could help eradicate genetically-based diseases like Alzheimer's and HIV.

However, as our reporters Naibe Reynoso and Valrie Defertexplain, this technique isbecoming available to the public even as it's still being tested for safety. Officials in the state of California are worried its use could get out of control.

An important application of genome editing is so-called gene drive, which could help put an end to malaria by altering the genomes of entire mosquito populations.

Our guest Dr. Jacob Corn,Professor of Genome Biology and Principal Investigator at the Corn Lab, ETH Zurichtells us how it could also be the solution to producing more food and doing so more efficiently to feed the world's growing population.

More here:
Tech 24 - CRISPR-Cas9: The era of genome editing - FRANCE 24

The CRISPR and CRISPR-Associated (Cas) Genes market research encompasses an exhaustive analysis of the market outlook, framework, and socio-economic impacts. The report covers the accurate investigation of the market size, share, product footprint, revenue, and progress rate. Driven by primary and secondary researches, the CRISPR and CRISPR-Associated (Cas) Genes market study offers reliable and authentic projections regarding the technical jargon.

All the players running in the global CRISPR and CRISPR-Associated (Cas) Genes market are elaborated thoroughly in the CRISPR and CRISPR-Associated (Cas) Genes market report on the basis of proprietary technologies, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, the report examines R&D developments, legal policies, and strategies defining the competitiveness of the CRISPR and CRISPR-Associated (Cas) Genes market players.

Request Sample Report @ https://www.tmrresearch.com/sample/sample?flag=B&rep_id=1250&source=atm

Trends and Drivers

The products available in the global CRISPR and CRISPR-associated (Cas) genes market are DNA-free Cas and vector-based Cas. The widening applications of these are expected offer several lucrative opportunities to the global market. Out of various applications, genome engineering is expected to be a key contributor to the soaring revenue of the overall market in the near future. This trend will be attributable to eh increasing uptake of genome editing method for the therapeutic development and germline modifications. The report indicates that advancements in plant genome engineering will result in positive impact on the global market.

Analysts predict that CRISPR could be the next biotechnology treatment that has the ability to gradually replace the present single-antibody drugs. Genome engineering is anticipated to pick up a phenomenal pace in the coming years as it is being developed to build an immune response for targeting cancer. The widening application of these methods in the field of oncology is likely to change the game for the global market in the coming years.

Global CRISPR and CRISPR-Associated (Cas) Genes Market: Regional Outlook

In terms of geography, the global market is segmented into North America, Asia Pacific, Latin America, the Middle East and Africa, and Europe. North America is estimated to lead the global CRISPR and CRISPR-associated (Cas) genes market as the U.S. has shown a keen interest in developing effective therapeutics. Asia Pacific is also expected to offer several growth opportunities to the overall market as the region is facing a challenge of mounting unmet medical needs.

Key Players Mentioned in the Report are:

The report has identified the following as the key operating players in the global CRISPR and CRISPR-associated (Cas) genes market: Thermo Fisher Scientific, Inc., Caribou Biosciences, Inc., CRISPR THERAPEUTICS, Addgene, Mirus Bio LLC, Merck KGaA, Editas Medicine, GE Healthcare Dharmacon Inc., Takara Bio USA, Horizon Discovery Group plc, and Intellia Therapeutics, Inc. Analysts predict that these companies will focus on making strategic collaborations to ahead of the competition present in the overall market.

Request For Discount On This Report @ https://www.tmrresearch.com/sample/sample?flag=D&rep_id=1250&source=atm

Objectives of the CRISPR and CRISPR-Associated (Cas) Genes Market Study:

The CRISPR and CRISPR-Associated (Cas) Genes market research focuses on the market structure and various factors (positive and negative) affecting the growth of the market. The study encloses a precise evaluation of the CRISPR and CRISPR-Associated (Cas) Genes market, including growth rate, current scenario, and volume inflation prospects, on the basis of DROT and Porters Five Forces analyses. In addition, the CRISPR and CRISPR-Associated (Cas) Genes market study provides reliable and authentic projections regarding the technical jargon.

Customize This Report @ https://www.tmrresearch.com/sample/sample?flag=CR&rep_id=1250&source=atm

After reading the CRISPR and CRISPR-Associated (Cas) Genes market report, readers can:

Go here to read the rest:
Promising Opportunities in North America and Europe to Propel the Growth of the CRISPR and CRISPR-Associated (Cas) Genes Market 2017 2025 Dagoretti...

The latest version of the 2020 market study on E waste Disposal Market comprising 111 with market data Tables, Charts, Graphs, and Figures which are easy to understand with showcased in-depth analysis.

The global E-waste Disposal market size is estimated at xxx million USD with a CAGR xx% from 2015-2020 and is expected to reach xxx Million USD in 2020 with a CAGR xx% from 2020 to 2025. The report begins from overview of Industry Chain structure, and describes industry environment, then analyses market size and forecast of E-waste Disposal by product, region and application, in addition, this report introduces market competition situation among the vendors and company profile, besides, market price analysis and value chain features are covered in this report.

Check out sample report at: https://www.amplemarketreports.com/sample-request/global-e-waste-disposal-market-1316664.html

As per the research and study, the market has settled its presence worldwide. E waste Disposal Market Research study offers a comprehensive evaluation of the Market and comprises a future trend, current growth factors, focused opinions, details, and industry certified market data.

Glancing to 2020, the global market expected to be a significant year for E waste Disposal Market in terms of growth and revenue.

Almost all companies who are listed or profiled are being to upgrade their applications for end-user experience and setting up their permanent base in 2020. This report focused and concentrate on these companies including (Company Profile, Sales Revenue, Price, Gross Margin, Main Products etc.), Sims Recycling Solutions, Eletronic Recyclers International, Kuusakoski, Umicore, Waste Management, Gem, Stena Metall Group, GEEP, Dongjiang, URT, Electrocycling, Cimelia, Veolia, Dynamic Recycling, Enviro-Hub Holdings, E-Parisaraa, environCom, Sage, IRT, Global Electronic Recycling.

Furthermore, the research contributes an in-depth overview of regional level break-up categorized as likely leading growth rate territory, countries with the highest market share in past and current scenario. Some of the geographical break-up incorporated in the study are 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.).

With the E waste Disposal market forecast to expand CAGR% in 2020 and with X-X-X-X supposed to be a big beneficiary, it is better positioned than Z-Z-Z-Z for 2020.

According to the AMR market study, Recent trends in consumer preferences market segments such as type, application will be more challenging. E waste Disposal market segment sales will traverse the $$ mark in 2020.

Unlike classified segments successful in the industry such as by Type (Infocomm technology (ICT) equipment, Home appliances) and by End-Users/Application (Material Recycling, Components Recycling).

The 2020 version of the E waste Disposal market study is a further split down / narrowed to highlight the latest emerging twist of the industry.

Enquire more before buy at: https://www.amplemarketreports.com/enquiry-before-buy/global-e-waste-disposal-market-1316664.html

Due to a change in consumer preferences with a review on the latest sales and revenue report submissions, Major vendors in the Global market are trying to get the attention of end-users or consumers by Offerings and additional services.

With using the latest technology and analysis on demand-side, Key players are getting in consumer behavior and their changing preferences.

Again, big investment firms or giants are willing to put more capital to get a key players performance in the market for new applications or products.

Discount, Know more this research report at: https://www.amplemarketreports.com/discount-request/global-e-waste-disposal-market-1316664.html

Research Objectives and Purpose

Read More about this report at: https://www.amplemarketreports.com/report/global-e-waste-disposal-market-1316664.html

Thanks for reading this article, you can also get individual chapter wise section or region wise report versions like North America, Western / Eastern Europe or Southeast Asia.

With the given market data, Research on Global Markets offers customizations according to specific needs. Write to AMR at [emailprotected], or connect via +1-530-868-6979

About Author

Ample Market Research provides comprehensive market research services and solutions across various industry verticals and helps businesses perform exceptionally well. Our end goal is to provide quality market research and consulting services to customers and add maximum value to businesses worldwide. We desire to delivery reports that have the perfect concoction of useful data. Our mission is to capture every aspect of the market and offer businesses a document that makes solid grounds for crucial decision making.

Contact Address:

William James

Media & Marketing Manager

Address: 3680 Wilshire Blvd, Ste P04 1387 Los Angeles, CA 90010

Call: +1 (530) 868 6979

Email: [emailprotected]

https://www.amplemarketreports.com

Read the original post:
CRISPR & CRISPR-associated (Cas) Genes Market Growth Opportunities and Forecast to 2027 - Fusion Science Academy

The market is estimated to grow with a CAGR of 17. 2% from 2018-2025. The growth of the genome editing market is primarily attributed to the rise in the production of genetically modified crops and rising prevalence of the genetic diseases.

New York, Jan. 24, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "North America Genome Editing Market to 2025 - Regional Analysis and Forecasts by Technology, Application End User, and Country" - https://www.reportlinker.com/p05774528/?utm_source=GNW However, the stringent regulatory framework and limitations in genome editing are likely to pose a negative impact on the market growth.

On the other hand, emerging markets for precision and regenerative medicines is likely to have a positive impact on the growth of the North America genome editing market in the coming years.The genome editing has proved itself to be the most promising way of feeding the fast growing population across the world.The changes in the climatic conditions due to the global warming and others conditions such as droughts floods are witnessed more across the world.

Therefore, the feeding the rising population is question among the people across the world.Due to the genome editing the concerns are being reduced to a great level, the two types of the genetically modified crops are widely grown.

Firstly, these crops are altered in a ways that they are not affected by the herbicide glyphosate.Secondly, crops are produced to protect themselves from the insecticides.

The advantages of the genetically modified crops includes diseases resistance, improvement of the photosynthesis, improvement of the nutrition, and more. The genetic modification helps to enhance the productivity without hampering the health of the crops. In addition, for the genetically modified crops the limited resources are required and it require less or no pesticides for its growth. The time required for the growth of the genetically modified crops is less, therefore these are highly preferred crops in the western world. The demand for the genetically modified crops is rising in the eastern region due to the benefits offered by these crops.According to the International Service For The Acquisition Of Agri-Biotech Applications (ISAAA), 2017 statistics, 19 developing countries have planted 53% which is approximately to 100.6 million hectares of the global biotech hectares, whereas the 5 industrial countries have took the 47% which is near about 89.2 million hectares share. The trend of growing genetically modified crop is expected to grow in the coming future.In 2017, the CRISPR segment segment held a largest market share of 53.6% of the genome editing market, by technology. This segment is also expected to dominate the market in 2025 owing to the simple, fast and accurate property of the CRISPR. Moreover, the TALENs segment is anticipated to witness the significant growth rate of 17.1% during the forecast period, 2018 to 2025 owing to the properties provided by the TALENs the market for it is expected to rise in the coming near future.North America genome editing market, based on application was segmented into genetic engineering, cell line engineering and others. The cell line engineering segment is anticipated to grow at a CAGR of 18.0% during the forecast period. Moreover, the genetic engineering segment is expected to grow at the significant rate during the coming years owing to its sub segments such as animal genetic engineering and plant genetic engineering that are being carried out extensively. In 2017, the biotechnology & pharmaceutical companies segment held a largest market share of 61.2% of the genome editing market, by end user. This segment is also expected to dominate the market in 2025 owing to the advantages of the CRISPR, the companies have enhanced their research and development for the drug discoveries that can treat various diseases. Hence, the market is likely to propel in the coming years.Some of the major primary and secondary sources for genome editing included in the report are, Contract Research Organizations (CRO), United States Department of Agriculture (USDA), National Institutes of Health (NIH), Abu Dhabi Fund for Development (ADFD), Ministry of Science and Technology (MST), International Service For The Acquisition Of Agri-Biotech Applications (ISAAA), Food and Drug Administration (FDA), Department of Biotechnology (DBT) and others.Read the full report: https://www.reportlinker.com/p05774528/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

Story continues

Clare: clare@reportlinker.comUS: (339)-368-6001Intl: +1 339-368-6001

See original here:
The North America genome editing market is expected to reach US$ 4,148.1 Mn in 2025 from US$ 1,234.5 Mn in 2017 - Yahoo Finance