Archive for the ‘Crispr’ Category

Portfolio manager Cathie Wood is known for having an aggressive appetite for risk when it comes to the investments she makes in her exchange-traded fund, the ARK Innovation ETF (ARKK -2.47%). Between its stakes in biotech companies with no products on the market and in rising stars like Tesla, its holdings are often in long shots that have the potential to be transformative for their industries or for the world.

On that note, there are two promising -- but speculative -- biotechnology businesses in the ARK portfolio that investors might be interested in if they're patient enough to hold onto their shares for a few years before seeing major returns. Over the next five years, Wood's thesis for both will be tested, so people who buy shares now could join in her eventual profits -- or her losses, which have been substantial for both stocks over the last 12 months.

CRISPR Therapeutics (CRSP 1.90%) is a gene-editing biotech that is working to develop cures for hereditary conditions including sickle cell disease and beta-thalassemia. Before the end of 2022, it expects to ask regulators at the Food and Drug Administration to approve the gene therapy exa-cel, which it claims can functionally cure both diseases. That means sometime in 2023, it could be realizing revenue from sales of its treatments for the first time ever, which will be a major catalyst for the stock.

But CRISPR's true potential actually lies beyond exa-cel. It's also investigating a handful of candidates as off-the-shelf immunotherapies that could treat different cancers, among them lymphoma. The off-the-shelf aspect is what differentiates these programs from the immunotherapies in development by most other companies, and it's also the most exciting thing about CRISPR.

A common problem with sophisticated cell therapies made using genetic engineering is that the patient's body may reject the cell therapy upon infusion. To get around that issue, biopharmas use each patient's own cells as the starting material to manufacture their specific therapy. That's effective, but it's also tremendously expensive because of the costs involved with drawing a sample, shipping it to a manufacturing site, processing it to make a single person's therapy, and then shipping it back to be infused into the patient.

What CRISPR hopes to do is produce non-personalized immunotherapies that don't trigger rejection. If it succeeds, its treatments will be far more scalable to manufacture, far more profitable to sell, and maybe even more effective than those produced by its competitors. That possibility is exactly what Wood is betting on with her investment, but it's almost certain to take a bit longer than a year or two to come to fruition because of how ambitious the goal is.

If you're willing to take a risk that CRISPR won't ever be able to figure it out for the chances of outsized rewards if it does, this is a good stock to buy.

Intellia Therapeutics (NTLA -1.48%) also plans to use advanced gene-editing techniques to treat people's genetic diseases. Like CRISPR Therapeutics, it doesn't have any revenue outside of what it makes from collaborations -- and that only totaled around $33 million in 2021. In terms of its pipeline, it has an early-stage program for sickle cell disease, and other early-stage programs aiming to address transthyretin amyloidosis, a rare hereditary liver disease.

Excitingly, its therapies for those conditions could be curative, though management is careful to remind investors that those treatments could still deliver much-needed relief to patients for long periods without being complete cures, technically speaking. But it isn't anywhere close to commercializing any of its therapies, so it's definitely a stock you'll need to hold onto for at least three or four years before it has the possibility of delivering major returns.

Furthermore, Intellia is developing capabilities similar to CRISPR's with regard to off-the-shelf immunotherapies, though CRISPR's are further advanced. It's pretty clear that Wood bought into Intellia to diversify her bet on easily scalable immunotherapies and give herself two opportunities to succeed. It might make sense for you to do the same if you're looking to hedge your other gene-editing stock plays.

Alex Carchidi has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics, Intellia Therapeutics, and Tesla. The Motley Fool has a disclosure policy.

Read more from the original source:
2 Risky Cathie Wood Growth Stocks to Buy and Hold for 5 Years - The Motley Fool

New Jersey, United States, Sept. 18, 2022 /DigitalJournal/ The mRNA Vaccines & Therapeutics Market research report provides all the information related to the industry. It gives the markets outlook by giving authentic data to its client which helps to make essential decisions. It gives an overview of the market which includes its definition, applications and developments, and technology. This mRNA Vaccines & Therapeutics market research report tracks all the recent developments and innovations in the market. It gives the data regarding the obstacles while establishing the business and guides to overcome the upcoming challenges and obstacles.

mRNA vaccines work by introducing a piece of mRNA that matches a viral protein, usually a small piece of a protein found on the outer membrane of the virus. Using this mRNA pattern, the cells produce the viral protein. RNA-based therapies have attracted a lot of attention in recent years due to their high potential in treating chronic diseases. Additionally, in terms of production, distribution and safety, RNA vaccines offer a number of advantages over DNA vaccines. They have also shown promise in human clinical studies, increasing the demand for mRNA-based vaccines and therapeutics.

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Competitive landscape:

This mRNA Vaccines & Therapeutics research report throws light on the major market players thriving in the market; it tracks their business strategies, financial status, and upcoming products.

Some of the Top companies Influencing this Market include:CRISPR Therapeutics, Precision NanoSystems, In-Cell-Art, GlaxoSmithKline Vaccines, ETheRNA immunotherapies, Roche Custom Biotech, PhaseRx, BioNTech, MaxCyte, Ethris, Kernal Biologics, AstraZeneca, Argos Therapeutics, RaNa Therapeutics, CureVac, Bayer, Intellia Therapeutics, Novartis, Janssen, Boehringer Ingelheim, Moderna Therapeutics,

Market Scenario:

Firstly, this mRNA Vaccines & Therapeutics research report introduces the market by providing an overview that includes definitions, applications, product launches, developments, challenges, and regions. The market is forecasted to reveal strong development by driven consumption in various markets. An analysis of the current market designs and other basic characteristics is provided in the mRNA Vaccines & Therapeutics report.

Regional Coverage:

The region-wise coverage of the market is mentioned in the report, mainly focusing on the regions:

Segmentation Analysis of the market

The market is segmented based on the type, product, end users, raw materials, etc. the segmentation helps to deliver a precise explanation of the market

Market Segmentation: By Type

Standardization Of Cancer Treatment MRNA VaccineIndividualized Cancer Treatment MRNA VaccineInfectious Disease Treatment MRNA VaccineInfection Prevention MRNA Vaccine

Market Segmentation: By Application

HospitalClinicOthers

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An assessment of the market attractiveness about the competition that new players and products are likely to present to older ones has been provided in the publication. The research report also mentions the innovations, new developments, marketing strategies, branding techniques, and products of the key participants in the global mRNA Vaccines & Therapeutics market. To present a clear vision of the market the competitive landscape has been thoroughly analyzed utilizing the value chain analysis. The opportunities and threats present in the future for the key market players have also been emphasized in the publication.

This report aims to provide:

Table of Contents

Global mRNA Vaccines & Therapeutics Market Research Report 2022 2029

Chapter 1 mRNA Vaccines & Therapeutics Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global mRNA Vaccines & Therapeutics Market Forecast

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mRNA Vaccines & Therapeutics Market to Witness Growth Acceleration | CRISPR Therapeutics, Precision NanoSystems, In-Cell-Art - Digital Journal

Biotech companies with few products on the market and red ink on the bottom line may not look like the most attractive investments right now. Equity markets are still down substantially year-to-date, and in this environment investors prefer putting their money in safer stocks.

But for those with a long-term mindset -- and above-average risk tolerance -- relatively small but promising biotech stocks can be compelling options to buy and hold. Here are two biotech stocks to consider buying that could make investors richer in time: Axsome Therapeutics (AXSM -2.56%)and CRISPR Therapeutics (CRSP 1.90%).

AXSM data by YCharts

Axsome Therapeutics is fresh off an important regulatory milestone. In August, The company's therapy for major depressive disorder (MDD), Auvelity, earned approval from the U.S. Food and Drug Administration (FDA). Auvelity took a while to finally earn the green light due to deficiencies in Axsome's application.But now that it's ready to hit the market, it could become highly successful.

The number of people with depressive symptoms increased threefold at the start of the pandemic, a trend that has persisted. As of 2021, more than 80 million people in the U.S. experienced symptoms of depression. In clinical trials, patients taking Auvelity saw substantial improvement compared to those taking a placebo. What's more, the medicine works relatively fast, with results as early as one week.

The long-term opportunities for this medicine are exciting, especially considering potential label expansions. Axsome recently started a phase 3 clinical trial for Auvelity in treating Alzheimer's disease (AD) agitation. Patients suffering from agitation show signs of emotional distress and physical aggressiveness.There were six million (and growing) AD patients in the U.S. in 2020, and 70% of them suffered from agitation.

Further, there are no approved treatments for this condition, which shows the potential Auvelity has in this area.

Axsome is also running a phase 2 study for Auvelity in smoking cessation treatment. The biotech has other promising programs, including AXS-07, a potential treatment for migraines. The FDA declined to approve AXS-07 earlier this year, but that was due to manufacturing issues, not problems related to the medicine's safety or efficacy. That means there is still an excellent chance AXS-07 will win approval.

Given that more than 37 million patients in the U.S. suffer from acute migraines -- and more than 70% of them are not satisfied with current treatments -- AXS-07's potential also looks strong. Axsome's lineup features Sunosi, a therapy for excessive daytime sleepiness in narcolepsy patients. And in 2023, the company expects to submit an application to the FDA for AXS-14 in treating fibromyalgia (a condition whose symptoms include excessive fatigue and musculoskeletal pain).

Axsome's lineup should become even more impressive within the next few years. The company reported about $8.8 million in sales from Sunosi -- and in total -- during the second quarter. The biotech had no revenue in the second quarter of 2021. Meanwhile, its net loss of $41.4 million was slightly worse than the net loss of $32.3 million reported during the year-ago period.

Axsome Therapeutics is in an excellent position to improve its financial results now that its research efforts are paying off. Expect the biotech company to gain in prominence and size in the coming years.

CRISPR Therapeutics focuses on gene-editing therapies. The company's lead candidate is exa-cel (formerly known as CTX001), a potential treatment for two rare blood diseases: sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). CRISPR has made some excellent decisions in its effort to market exa-cel. Most of all, it partnered with biotech giant Vertex Pharmaceuticals.

True, CRISPR Therapeutics will have to share the profits associated with exa-cel with its partner on this program if it gets approved. But clinical-stage biotechs are often strapped for cash, and developing innovative therapies isn't cheap. Further, whether an investigational treatment will earn approval is never a sure thing, no matter how promising it looks.

Regardless of whether exa-cel is approved, CRISPR Therapeutics has already collected payments from Vertex in exchange for sharing the rights to the therapy -- the former will receive 40% of the profits and incur 40% of the costs.

The most impressive thing, though, is that exa-cel has been able to produce excellent results in clinical trials to treat these two rare illnesses which have seriously challenged researchers. For instance, 42 of 44 TDT patients treated with the gene-editing therapy were transfusion-free, with follow-up ranging between 1.2 months and 37.2 months.The two that were not yet transfusion-free experienced substantial reductions in transfusion volume.

Vertex Pharmaceuticals and CRISPR Therapeutics plan on filing regulatory applications with the appropriate authorities in the U.S. and Europe by the end of the year. Potential launches could come in late next year. CRISPR also boasts other pipeline candidates. The company's gene-editing platform will get a major vote of confidence if exa-cel earns approval. Of course, it will work wonders for its financial results, too.

The biotech's status as a clinical-stage biotech that is not yet profitable does not inspire much confidence at first glance. But CRISPR Therapeutics could end up being an excellent pick for patient investors.

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2 Biotech Stocks That Could Make You Richer - The Motley Fool

FRONT PAGE

Indian and Chinese troops complete disengagement in Hot Springs region

Syllabus:

Preliminary Examination:Current events of national and international importance.

Mains Examination:General Studies II: India and its neighbourhood- relations.

Key Points to Ponder:

Whats the ongoing story- Indian and Chinese troops have completed the disengagement process at Patrolling Point-15 in the Gogra-Hot Springs region of eastern Ladakh, sources in the military establishment said on Tuesday.

Know the Background-In May 2020 when China had diverted its troops who had come to the Tibetan plateau region for their annual exercise, towards the Line of Actual Control (LAC) in eastern Ladakh, creating a standoff with India, PP15 and PP17A were two of the four points where the soldiers were eyeball-to-eyeball.

The six-day process had five components-what are they?

What are PP15 and 17A?

For Your Information-PP15 is located in an area known as the Hot Springs, while PP17A is near an area called the Gogra post.

Map Work-Chang Chenmo river, Gogra-Hot Springs, Kongka Pass, Galwan Valley, Depsang Plains, Line of Actual Control, and Charding Nala region

What is the importance of this region for India?

How significant are they for the military?

India-China Relations during Nehruvian Era-Know in detail

The 1962 India-China War-Know the background

India-China Border Dispute- Know the background

What is Line of Actual Control?

Chinas aggressive attitude towards Indo-China Border and What impact can it have on India-China relations?

Changing dynamics in Indo-China relationships-what are the points of irritation in recent scenario?

Jingoism and not pragmatism nowadays dominate bilateral relations of India with her Neighbours -do you think so? Attest your opinion with few examples

Resolving the Sino-Indian imbroglio-How?

Other Important Articles Covering the same topic:

Explained: In India-China border dispute, strategic significance of Hot Springs, Gogra Post

Dealing with China

Disengagement on Line of Actual Control is a welcome start, but normalisation of India-China relations is a long way off

Govt confirms: Delhi to host G20 summit next Sept

Syllabus:

Preliminary Examination:Current events of national and international importance.

Main Examination:General Studies II: Bilateral, regional and global groupings and agreements involving India and/or affecting Indias Interests

Key Points to Ponder:

Whats the ongoing story India will host the G-20 leaders summit in New Delhi on September 9 and 10 in 2023 under its Presidency, the Ministry of External Affairs (MEA) announced Tuesday.

For your Information India will assume the Presidency of the G20 for one year from December 1, 2022, to November 30, 2023, and is expected to host over 200 meetings across the country, beginning in December this year. India, as G20 Presidency, will be inviting Bangladesh, Egypt, Mauritius, Netherlands, Nigeria, Oman, Singapore, Spain and UAE as Guest countries, said the MEA.

Do you Know-During our Presidency, India, Indonesia and Brazil would form the troika. This would be the first time when the troika would consist of three developing countries and emerging economies, providing them a greater voice, said the MEA in a statement.

What is G20?

Know the origin of G20

How G20 Works?

G20 or Group of Twenty-About, Purpose and Member Countries

What is G20 Troika?

G20 Troika and India-Know in detail

Procedure for taking over the G20 presidency-How it is Decided?

G20-Relevance in todays Changing Geopolitical Dynamics?

For Your Information-Collectively, the G20 accounts for 85 per cent of global GDP, 75 per cent of international trade and two-thirds of the world population, making it the premier forum for international economic cooperation. India is currently part of the G20 Troika (current, previous and incoming G20 Presidencies) comprising Indonesia, Italy and India.

Map Work-G20 member Countries

Other Important Articles Covering the same topic:

Explained: What is the G20, of which India becomes president later this year?

GOVT & POLITICS

Amendment for EWS quota is fraud on Constitution, SC told

Syllabus:

Preliminary Examination:Indian Polity and Governance

Main Examination:General Studies II: Government policies and interventions for development in various sectors and issues arising out of their design and implementation

Key Points to Ponder:

Whats the ongoing story-Questioning submissions that 103rd Constitution amendment provides reservation to Economically Weaker Sections (EWS) without any guard rails unlike in the case of reservations for backward classes, where conditions like maintenance of efficiency of administration is prescribed in Article 335 the Supreme Court on Tuesday said the Constitution did not provide for any such guard rails even in the case of reservation for women.

What Supreme Court said about Article 15(3) part of the original Constitution?

Social justice is the first objective of the Constitution and the heart and soul of the Republic-How Constitution of India ensures social justice?

Economically Weaker Sections and Socially Weaker Sections-Compare and Contrast

What is the 103 Amendment in Indian Constitution?

Know the key highlights of the Economically Weaker Sections Reservation-103rd Constitution (Amendment) Act, 2019

What was the Supreme Courts Verdict in Indira Sawhney case 1992?

Supreme Court on EWS Reservation Criteria-Know about it

EWS Reservation-Issues and Challenges

EWS quota: What are the issues fixed by the Supreme Court?

How is EWS status determined under the law?

What is the basis of the challenge to the amendment?

What has been the governments stand in this matter so far?

What is the difference between quota and reservation?

Other Important Articles Covering the same topic:

Explained: Revisiting definition of EWS

List of essential drugs updated; new diabetes, anti-cancer drugs added

Syllabus:

Preliminary Examination:Current events of national and international importance.

Mains Examination: General Studies II: Government policies and interventions for development in various sectors and issues arising out of their design and implementation.

Key Points to Ponder:

Whats the ongoing story-The Union Health Ministry on Tuesday launched the new National List of Essential Medicines (NLEM), expanding the list to include newer therapies for diabetes, such as the medicine Teneligliptin and the insulin Glargine, and also incorporating four more anti-cancer therapies.

For Your Information-The NLEM guides the governments procurement policy and decides the price cap for medicines. The updated list has deleted 26 drugs from the previous one and added 34 drugs, increasing the list to 384 drugs.

What is National List of Essential Medicine (NLEM)?

Do You Know-The NLEM was first formulated in 1996 and was revised in 2003, 2011, and 2015. It takes into account any changing profile of diseases, newer drugs available in the market, and changing treatment protocols. The price of medicines in the list is controlled by the Centre and cannot be changed by companies themselves. Many of these medicines are also available free at government health facilities.

National List of Essential Medicine (NLEM) in India-Know in detail

Who is National Pharmaceutical Pricing Authority (NPPA)?

National Pharmaceutical Pricing Authority (NPPA) and National List of Essential Medicine (NLEM)-Connect the dots

Why National List of Essential Medicine (NLEM) is significant?

Other Important Articles Covering the same topic:

In a first, regulator hikes prices of essential medicines

EXPRESS NETWORK

Cheetah mitras to watch towers, Kuno ready to host African guests

Syllabus:

Preliminary Examination:General issues on Environmental ecology, Bio-diversity and Climate Change

Main Examination:General Studies III: Conservation, environmental pollution and degradation, environmental impact assessment.

Key Points to Ponder:

Whats the ongoing story- Equipped with a small shed for shade and a few trees, a 5030-metre quarantine enclosure is already at Madhya Pradeshs Kuno National Park to host eight cheetahs arriving from Namibia. Prime Minister Narendra Modi will release three cheetahs two male siblings and a female into the enclosure Saturday to launch the re-introduction of the species in India.

Cheetah in India- Background

Who are cheetah mitras?

Extinction of Cheetah from Indian Landscape-know the reasons

What is the Reintroduction of the cheetah in India plan?

How Reintroduction of the cheetah in India plan is executed?

Action Plan for Introduction of Cheetah in India-Important Highlights

Know the difference between cheetah and Leopard and African cheetah and Asiatic cheetah

Know the Difference between Extinct, Extinct in the Wild and Critically Endangered

Supreme Court of India on Translocating Animals

Translocating Animals-Issues and Challenges

View post:
UPSC Key-September 14, 2022: Why you should read 'National List of Essential Medicine' or 'CRISPR Technology' or 'Coastal Regulation Zone' for UPSC...

In March 2011, a chance meeting between two women changed history without anyone being aware of it. It happened in a cafe in San Juan, Puerto Rico, when the American molecular biologist Jennifer Doudna was introduced to Emmanuelle Charpentier, a French professor of Armenian origin in the same field. They hit it off and, within hours, agreed to a research collaboration. The result, a year later, was the discovery of the CRISPR system: a technology that can be used to edit genes.

Eleven years later, CRISPR is a ubiquitous tool in any molecular biology laboratory in the world, allowing research to be carried out at a speed and cost that were previously unimaginable. Gene editing has also made its way into the experimental treatment of many diseases. CRISPR makes mind-blowing applications possible, such as taking white blood cells from a person, rewriting their genomes to transform them into cancer-killing machines and reinjecting them to fight tumors that havent responded to conventional treatments.

Charpentier and Doudna won the 2020 Nobel Prize in Chemistry for discovering CRISPR. Charpentier, 53, made her fundamental contribution while searching for a way to kill an implacable enemy: the streptococcus pyogenes bacteria, one of the top 10 causes of deadly infections on the planet. It is known as the flesh-eating bacteria because of the horrible wounds it causes if it gets under the skin and reaches the muscle a type of injury that has been documented since the 5th century, appearing in terrible situations. For instance, it inflicted combatants in the US Civil War and heroin addicts in 1990s San Francisco. This bacteria has even become immune to conventional antibiotics.

The investigation into the molecular mechanisms that this bacterium uses to survive external threats was key to discovering CRISPR. The microbes acted as a kind of bacterial immune system, capable of remembering precise fragments of the virus genome, to then cut the virus DNA. But the genome of a virus has millions of letters arranged one after the other how did these microbes manage to identify the genome of the virus and cut it in the exact place?

A few months before the historic meeting in Puerto Rico, Charpentiers team had discovered an RNA molecule that was essential to guiding a pair of molecular scissors to the exact sequence of the genome of each virus. This was key to putting together all the elements needed to build the new CRISPR gene-editing tool.

Three months ago, CRISPR Therapeutics the company Charpentier founded in 2019 published preliminary results from a clinical trial showing that 15 patients with beta thalassemia a severe type of anemia that requires lifelong reliance on blood transfusions had gone months without needing them after receiving a drug that edited the gene that caused the disease.

In early September, Charpentier traveled to Yerevan, Armenia to be one of the main speakers at the Starmus VI Festival. In her interview with EL PAS, the scientist explains that she is still focused on the same goal as she was years ago: looking for new forms of gene editing to combat antibiotic-resistant infections. These superbugs already kill more people than AIDS, malaria and some cancers. For her, one of the greatest dangers we face is that the basic sciences which require years of hard work are no longer attractive to young people, who will need to invent new treatments and medicines in the future. This interview has been edited for the purposes of clarity and brevity.

Question. Where did your interest in science come from?

Answer. At the age of 15, I was obsessed with monasteries I wanted to be a nun for a while. Thats maybe reflected in my work as a scientist: many hours are spent alone, cut off from the world. This is what I did at the University of Ume, in Sweden. I made the key discovery for CRISPR there, while living in my scientific monastery in northern Sweden. At the same time, I was very interested in detective stories, searching for enigmas.

Q. Do you believe in God?

A. My parents were Catholics, but they belonged to a very modern and up-to-date branch of Catholicism, with working-class priests. I grew up in this environment and practiced, but I havent done it for a long time. For me, believing in God is believing in the good of the human being, the best version of humanity.

Q. Are microbes superior to humans?

A. Probably, yes. Long after we have disappeared from the planet, they will still be here. And lets not forget that they already existed long before we appeared! They have solved key problems in their own way. They know how to communicate, adapt, fight theyre extremely versatile. And were talking about a huge community, with millions of different species and an exciting social life.

Q. Social life?

A. They are very social. We can learn a lot from them. The human body contains more bacterial cells than human cells. And this community partly determines how we react to stimuli, why we get sick, how our metabolism works, even some brain functions. I believe that the challenge for human beings is to adapt to the enormous change that is taking place in the microbial universe. We have seen it with a single microbe: SARS-CoV-2. And we are going to see it with new viruses that are yet to come. Many of them will, in part, be driven by human activities on the planet.

Q. Just four years ago, when you spoke to EL PAS, there were hardly any applications of CRISPR in health. Now, especially after the pandemic, there are more and more. Whats the next big step?

A. In origin, CRISPR is an immune system that allows bacteria to defend themselves against viruses. In the coming years, we must perfect the system and be able to use it in a more personalized way. Its the future the study of microbes can solve some of the biggest problems facing humanity. We can create crops that are more resistant to changes in climate and the environment. But the next big step as is usually the case in science will be totally unexpected.

Q. And what role will CRISPR play in treating disease?

A. This tool could help interfere with human metabolism in a beneficial way, to eliminate the negative effects of common diets in the Western world. These same problems are becoming more and more prevalent in Asia [the developing world], because the populations metabolism is not prepared for this type of diet, with lots of meat and enriched carbohydrates. One of the key ailments in this field would be diabetes, for example. Also obesity and infectious diseases.

Q. You head the Max Planck Institute for the Science of Pathogens (Berlin). One of its goals is to combat antibiotic-resistant infections, which are expected to cause the next pandemic.

A. My lab doesnt have many people. We continue to work in a humble way on the specific mechanisms existing in bacteria, so we can identify therapeutic targets and have new antibiotics ready to combat future infections. The development of antibiotics has stagnated in the last 20 years because the pharmaceutical industry is not interested in developing them. We are now starting to see small biotech companies tackling this challenge. I think it is very important to focus on this.

Q. Dont we already know how resistant bacteria are able to make us sick?

A. There are many different mechanisms. The problem is that, as soon as you create a new antibiotic, the bacteria develop immunity to it. Its important to continue researching in this field, looking for new ways to intervene and have different therapeutic compounds. A vaccine is one thing, but you also need antivirals. You have to have different strategies.

Q. What other future issues are you worried about?

A. Being well-armed against resistant bacteria requires a lot of work, a lot of research time. The efforts of many people in different fields, from biologists to doctors, entrepreneurs and businessmen. But the most indispensable are the basic scientists. With the noise of the world we live in today, we see many scientists who finish their doctorates and abandon research. Young generations cant find their place in the academic world, which has not evolved in 30 years. Meanwhile, everything is moving faster and faster, including business. In the US, you can create a biotech company almost instantly and be successful very quickly.

The basic sciences which rely on public funding are ceasing to be attractive, both in terms of funding and mentality. Young people do not want to wait so many years to see the fruits of their labor. Science is being flooded with politics. The system of scientific publications has been filled with marketing. If it continues like this, it will be a very serious problem. Scientists also teach at universities: they are the teachers of the next generations. Without them, not one, but several generations of brains can be lost.

Q. But more and better science is still being done, right?

A. I believe that fundamental biology and the basic sciences overall are in danger. Science involves isolating yourself and working very hard. You have to be able to read more than two pages at a time and work more than eight or nine hours at a stretch. We now see that young students have more and more trouble concentrating or working long hours.

Q. Do you see a solution to this problem?

A. No. I think we all and especially young people need to ask ourselves what kind of world we want to live in. I think kids in rich countries are going to realize that, if they dont change their attitude, theyll be digging their own graves.

Follow this link:
Dr. Emmanuelle Charpentier: We all need to ask ourselves what kind of world we want to live in - EL PAS USA

Figure 3. tracrRNA directs pre-crRNA cleavage by RNase III in vitro

a, Schematic representation of

a, Schematic representation of tracrRNA89 corresponding to 89-nt long tracrRNA, and crRNA213 and crRNA148 corresponding to a 213-nt long leader-repeat-spacer1-repeat-spacer2 fragment and a 148-nt long spacer1-repeat-spacer2-repeat-spacer3 fragment, respectively. b, Identification of tracrRNA89 binding sites on crRNA148*. 5 end-labeled crRNA148* (~10 nM) was subjected to lead(II), RNase III and RNase T1 cleavage in the absence (lanes 1, 4, 7) and presence of cold tracrRNA89 (final concentration in lanes 2, 5, and 7: ~50 nM; lanes 3, 6, and 9: ~500 nM). Lane C: untreated crRNA148*; Lane T1: RNase T1 digest of crRNA148* under denaturating conditions; Lane OH: alkaline ladder; cleaved G residues are labeled. Vertical bars: crRNA148 region protected by tracrRNA89. Arrows denote specific RNase III cleavages in the two repeat regions of crRNA148 in the presence of tracrRNA89. c, Identification of crRNA148 and crRNA213 binding sites on tracrRNA89. 5 end-labeled tracrRNA89* (~10 nM) was subjected to RNase T1, lead(II) and RNase III cleavage in the absence (lanes 1, 6, 11) and presence of cold crRNA148 or crRNA213 (final concentration in lanes 2, 4, 7, 9, 12 and 14: ~50 nM; lanes 3, 5, 8, 10, 13 and 15: ~500 nM). Lanes C, T1 and OH, positions of cleaved Gs and vertical bars: as above but referring to tracrRNA89* in the presence of cold crRNA148 or crRNA213.

Read more here:
CRISPR RNA maturation by trans-encoded small RNA and host ... - PubMed

CRISPR is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of viruses called bacteriophages that had previously infected the prokaryote. They are used to detect and destroy DNA from similar bacteriophages during subsequent infections, providing the prokaryote with a sort of immunity.

The following is an interview with CRISPR co-discoverer and Nobel Prize-winner Dr. Jennifer Doudna.

Describe the eureka moment around CRISPR the moment when you realized that this technology was not only possible but actually worked. How did you feel? Has your feeling changed since that eureka moment? If so, how?

Theres one moment that stands out in my mind, right at the time we realized what CRISPR could do and that we could reprogram it to edit specific sequences of DNA. I was cooking dinner and thinking about it, and I burst out laughing. My son was in the kitchen and he asked why I was laughing. So I explained it to him with a little drawing of a car zooming around, grabbing onto viruses, and chopping them up. I think my drawing did the trick, because he started laughing too.

The implications of this finding were too big to understand all at once. Its been ten years since that time now, and everything that has happened since surpassed any expectations I had back then. With multiple therapies in clinical trials, plants in fields that help farmers adapt to a changing climate, and countless uses of CRISPR in life science research, the scope of what has been achieved in just ten years continues to surprise me.

What excites and inspires you most about the possibilities of CRISPR technologies?

I recently spoke with Victoria Gray, one of the first people to receive a CRISPR-based therapy for sickle cell disease. Hearing from her about how her life has changed for the better, how shes no longer in constant pain and able to go back to work and spend more time with her family theres nothing more inspiring than real human impact. Thats what drives the work we do at the institute that I started at UC-Berkeley, the Innovative Genomics Institute (IGI), where the focus is not just developing new therapies and agricultural products, but making sure they reach the people who need them most.

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What is the most interesting, or counterintuitive, use of CRISPR technology that youve encountered thus far?

We talk a lot about the ability of CRISPR to cut DNA, but its ability to find a specific sequence of DNA is just as interesting. Thats not an easy thing to do, and it turns out that it can be really useful in other ways. For example, at the IGI, were developing CRISPR-based diagnostics for infectious disease. Instead of editing DNA, these tests quickly find a specific sequence of DNA from a pathogen, like the SARS-CoV-2 virus or HIV, and then release a fluorescent marker. The great thing about these tests is that theyre fast, can be performed anywhere, and should be quite cheap to produce. After everything weve all experienced during the pandemic, its clear that rapid point-of-need tests are going to be increasingly important.

Are there any parallels in history of a technology that fundamentally changed human life?

In many ways CRISPR genome editing builds on groundbreaking technologies and innovations that came before it, and each one was a watershed moment for science. We needed X-ray crystallography to understand the structure of DNA, Sanger sequencing to be able to read it, PCR to make copies of it, and the Human Genome Project and other large bioinformatics projects to start to understand the bigger picture of how genomes function. Being able to edit the genome is the next chapter in this story, but it couldnt exist without the others that came before it.

How can we most responsibly use the power this technology has unlocked? Where should we put the guardrails?

With any powerful technology, there is always potential for its misuse. And we have already seen this, even though the vast majority of scientists are using it responsibly. Determining what constitutes misuse, what is unethical, what is medically necessary that is where a lot of the discussion is focused at the moment. There is broad agreement on certain topics, particularly around human germline editing, but when it comes to questions of ethics, there will always be gray areas.

One risk that is often overlooked is the real possibility that some of the advances we make in genome editing will benefit a small fraction of society. With new technologies this is often the case at first, so we have to consciously work from the start to make new cures and agricultural tools that are accessible and affordable.

In your mind, what does it mean for humanity to have the ability to directly alter genetic material so precisely?

Its a powerful tool, and one that can be used to do a lot of good. Sickle cell disease affects millions of people worldwide, and its caused by a single-letter mutation in just one gene. This has been understood for a long time, but we didnt have the means to fix that mutation. There are several thousand other genetic diseases, including very rare diseases that are often neglected, that we can now look to address. It goes beyond medicine: Climate change is impacting agriculture, and agriculture itself is contributing to climate change. With genome editing, we can mitigate both of those impacts.

How do you think CRISPR will affect our understanding and definition of what it means to be human?

Understanding even just a little bit about genetic disorders what causes them, how many people are affected by them increases your compassion for what people are going through of no fault of their own. You also start to understand that there are people who have genetic mutations that affect their lives, but dont necessarily view them as diseases or problems to fix. CRISPR itself may not change what it means to be human, but perhaps having a tool that can rewrite our DNA helps to shine a light on all of the diversity that humanity already encompasses.

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10 years on, a spin-off use for CRISPR: Infectious disease testing - Big Think

The invasive Drosophila suzukii fruit fly has caused millions of dollars of damage to berry and other crops. Credit Michelle Bui, UC San Diego

Applying new CRISPR-based technology to a broad agricultural need, researchers at the University of California San Diego have set their aims on a worldwide pest known to decimate valuable food crops.

Nikolay Kandul, Omar Akbari and their colleagues first demonstrated the precision-guided sterile insect technique, or pgSIT, in Drosophila melanogaster, the common fruit fly, in 2019. The technology, later adapted to mosquitoes, uses programmable CRISPR techniques to edit key genes that control sex determination and fertility. Under the new system, pgSIT-developed insect eggs are deployed into a targeted population and only sterile males hatch, resulting in a fertility dead end for that species.

Kandul, Akbari and their colleagues have now adapted the technology for use in Drosophila suzukii, an invasive fruit fly (also known as the spotted-wing drosophila) responsible for millions of dollars in crop damage. The advancement is described as the cover paper in the journal GEN Biotechnology.

Former UC San Diego graduate student Stephanie Gamez created this artistic depiction of genetics and the fruit crop pest Drosophila suzukii.

Its a safe, evolutionary stable system, said Akbari, a professor in the School of Biological Sciences Department of Cell and Developmental Biology. Also, the system does not lead to uncontrolled spread nor does it persist in the environmentboth important safety features that will help it gain approvals for use.

D. suzukii flies have invaded many parts of the world and caused widespread agricultural and economic damage to several crops, including apples, cherries, raspberries, blueberries, strawberries, peaches, grapes, olives and tomatoes.

The flies are known to proliferate by depositing their eggs inside growing fruit. They are notoriously difficult to control since their larvae consume ripening fruit pulp, limiting the effectiveness of insecticide sprays. Some flies have been known to become resistant to insecticides and many chemicals used in insecticides are now banned because of threats to human health.

The concepts behind pgSIT date back to the 1930s, when farmers found ways to release sterile males into their crops to reduce damage from pests. By mid-century, United States farmers began using radiation to sterilize pests such as the New World screwworm fly.

With CRISPR, UC San Diego scientists avoided the need for harmful radiation and instead use CRISPR editing to specifically target genes essential for female D. suzukii viability and male fertility. As envisioned, pgSIT eggs could be produced at a factory and released at sites invaded by pests such as D. suzukii. Eggs could be deployed directly into areas where the flies are causing damage and only sterile males would hatch after about two weeks. Since only two genes are knocked out, the males emerge fit enough to compete with their wild counterparts and quickly seek females to mate with, resulting in inviable offspring.

Spotted-wing drosophila flies deposit their eggs inside strawberries and other ripening fruit. Credit Michelle Bui, UC San Diego

This technology would replace the need for insecticides and only suppress the target species population, said Akbari. In the last four years, weve developed pgSIT for several different species. Going forward were hoping to use it as a platform technology that can be ported to a whole range of pests to safely solve real-world problems.

Agragene Inc., a private biotechnology company co-founded by Akbari, has licensed the pgSIT base technology from UC Riverside (where Akbari initially led the technologys development) and is implementing U.S. Department of Agriculture-administered field trials of pgSIT in D. suzukii. The company hopes that the trials will demonstrate the safety and effectiveness of pgSIT and lead to regulatory approval of the technology for broad agricultural use.

The GEN Biotechnology paper was coauthored by: Nikolay Kandul, Junru Liu, Anna Buchman, Isaiah Shriner, Rodrigo Corder, Natalie Warsinger-Pepe, Ting Yang, Amarish Yadav, Maxwell Scott, John Marshall and Omar Akbari.

Support for the research came from the California Cherry Board, Washington Tree Fruit Board (19-CCB5400-06); Agragene (200779); the National Institute of Food and Agriculture; U.S. Department of Agriculture Specialty Crops Research Initiative (agreement No. 2015-51181-24252); and the Innovative Genomics Institute at UC Berkeley.

Note: Akbari is a co-founder with equity interest, and former consultant, scientific advisory board member and income recipient of Agragene Inc. Akbari and Kandul, who have submitted a patent application on pgSIT technology, are co-founders of Synvect Inc. with equity interest.

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CRISPR-based Technology Targets Global Crop Pest - University of California San Diego

The partnership will unlock access to CRISPR DNA editing technology and a consortium of scientists and resources to the thylacine de-extinction effort.

We can now take the giant leaps to conserve Australias threatened marsupials and take on the grand challenge of de-extincting animals we had lost, Professor Pask said.

A lot of the challenges with our efforts can be overcome by an army of scientists working on the same problems simultaneously, conducting and collaborating on the many experiments to accelerate discoveries. With this partnership, we will now have the army we need to make this happen.

Professor Pask said TIGGR will concentrate efforts on establishing the reproductive technologies tailored to Australian marsupials, such as IVF and gestation without a surrogate, as Colossal simultaneously deploy their CRISPR gene editing and computational biology capabilities to reproduce thylacine DNA.

Colossals resources and expertise in CRISPR gene editing the cutting and editing of DNA sequences to produce a genetic code to be developed into living organisms will be paired with TIGGRs work sequencing thylacine genome and identifying marsupials with similar DNA to provide living cells and template genome that can then be edited to recreate a thylacine genome.

The question everyone asks is how long until we see a living thylacine and Ive previously believed in ten years time we would have an edited cell that we could then consider progressing into making into an animal, Professor Pask said.

With this partnership, I now believe that in ten years time we could have our first living baby thylacine since they were hunted to extinction close to a century ago.

Colossal co-founder and CEO Ben Lamm said: We are thrilled to be collaborating with Andrew Pask and the University of Melbourne to restore this amazing animal to Earth while also further developing gestational and genetic rescue technologies for future marsupial conservation efforts.

Colossal Biosciences uses breakthrough gene-editing technologies to advance wildlife and ecosystem conservation and is also pursuing de-extinction of the woolly mammoth once the keystone species to the Arctic Tundra.

TIGGRS partnership with Colossal Biosciences will produce technology and knowledge to also influence the next generation of Australias marsupial conservation efforts and combat increasing extinction events caused by invasive species and climate change.

Our efforts to protect the endangered Northern Quoll long threatened by the invasive cane toad native to South and Central America - will also be aided by this partnership, as we could produce Northern Quolls with a slight genome-edit making them resistant to cane toads, giving Quolls the same evolutionary benefit of the many South and Central American animals resistant to cane toad-poison, Professor Pask said.

On the reproductive technology front, Professor Pask said TIGRR lab is also close to producing the first lab-created embryos from Australian marsupial sperm and eggs.

We are pursuing growing marsupials from conception to birth in a test-tube without a surrogate, which is conceivable given infant marsupials short gestation period and their small size.

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Lab takes 'giant leap' toward thylacine de-extinction with Colossal genetic engineering technology partnership - University of Melbourne

For years, Canadian artist Grimes was known for her music. But in the late 2010s, the Oblivion singer became known for making headlines, including having children named X A-Xii and Exa Dark Siderl with billionaire Elon Musk. Now, Grimes is looking to make body modifications to her teeth and her ears.

In August 2022, Grimes took to her Twitter account to ask fans if they know any good plastic surgeons who would be able to modify her appearance.

2 years ago I made an appointment with a great plastic surgeon, thought I might want to change things up by my mid-30s, but then I forgot and never thought about what I should do. Any face mods yall think would look good on me? she asked.

Also does anyone know anyone great/safe/reliable people who could do vampire teeth caps on me in Austin or LA? Also, any reputable elf ear modifiers in either of these cities? she said in another tweet, adding, Still debating this surgery cuz cartilage doesnt heal so it requires permanent stitches.

To ensure that what she was looking into was safe, she asked her followers if anyone had done ear modifications themselves. Has anyone done elf ear mods with a good outcome? she tweeted. Im scared about ear cartilage having a hard time healing. Especially as a musician this surgery seems risky but Ive wanted it my whole life. Curious about peoples experiences!

She then apologized for going through her thought process on social media. Sorry if weird to discuss this openly, just seems unhealthy how everyone in media hides body mods, then people feel self conscious, she tweeted. Im also less interested in conventional beauty (I will keep my nose) but moreso is there anything else that would look sick?

As for what the father of her two children thinks, Elon Musk weighed in by replying to her tweet about the ear modifications. The downside of elf ear surgery probably outweighs the upside, the Tesla CEO said.

Grimes responded with her own wishes to genetically modify her ears. This sounds like a job for CRISPR, she tweeted. Sad to be born just a few generations too early.

According to MIT and Harvards Broad Institute, CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology.

The term CRISPR, then, is used for systems that can be programmed to target specific stretches of genetic code and to edit DNA at precise locations, as well as for other purposes, such as for new diagnostic tools.

In addition to changing some of her facial and body features, Grimes is also busy working on new music.

She released her fifth album Miss Anthropocene in February 2020, months before giving birth to her first child. Shes set to released her sixth album, Book 1, sometime in 2022.

RELATED: Grimes Insists Shes Not a Communist After Reading Marxs Communist Manifesto Following Her Breakup With Elon Musk

Originally posted here:
Grimes Wants to Get Surgery to Have Elf Ears and Vampire Teeth - Showbiz Cheat Sheet

Editors note: WRAL TechWire today launches a new package of features focusing on innovation. This package will be part of Innovation Thursday.

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INNOVATION ROAD TRIP: Part One

Earlier this year, WRAL published a series titled Innovation Road Trip that featured six video and story packages from across North Carolina. Check out part one:

How CRISPR is solving problems through DNA editing

Written by Abbey Slattery, WRAL Digital Solutions

Eradication of certain diseases, increasing crop sizes, reducing pest populations the current and future applications of CRISPR have the potential to change ways of life around the world.

A tool for editing genomes including altering DNA and modifying gene functions CRISPR is short for CRISPR-Cas9 and refers to a string of DNA and the Cas9 enzyme. CRISPR works by finding a specific section of DNA, cutting it, then inserting a mutation or replacing a faulty gene.

Read the full story at this link.

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Innovation Thursday: How CRISPR is solving problems through DNA editing - WRAL TechWire

Cell and gene therapies are taking the world by storm. With CRISPR gene-editing technology, the genome is no longer an untouchable sacred text. Instead, genomic DNA is actively manipulated in the laboratory and the clinic. This revolution is changing the bench, the bedside, and the boardroom. As a result, the cell and gene therapy market is valued at USD 4.99 billion and is expected to grow to over USD 36 billionby 2027.

The potential of personalized medicinewhere treatments are specific to an individual's geneticsexplains this exponential growth. Cell therapies, such as CAR-Ts, modify cells outside the patient and reintroduce them as a targeted treatment. In comparison, gene therapies alter the DNA within cells. The final genetic dosage from a cell or gene therapy walks a razor's edge between therapeutic and toxic. This makes the quality control testing of cell and gene therapies absolutely vital. Many turn to qRT-PCR for this task, but this method cannot deliver absolute quantification as each run requires a standard curve, creating inconsistencies.

In this article, we explore how Droplet Digital PCR (ddPCR) technology is revolutionizing quality control for cell and gene therapies, enabling scientists to save time, money, and resources through confident quantification.

Before we dive into how ddPCR assays reimaginethe limits of quality control in cell and gene therapies, lets explore ddPCR technology. Like qRT-PCR, ddPCR technology requires a nucleic acid sample. However, in ddPCR assays, samples are partitioned into 20,000 discrete droplets. Individual PCR reactions occur within each droplet, which on average contains just a single copy of the donor DNA. During PCR cycling, positive droplets that contain the target DNA will produce a fluorescent signal. This digital system results in absolute quantification without the need for standard curves, leading to unparalleled precision, simplified workflows, and removal of PCR bias.

Generating cell and gene therapies is complex, requiring precise quality control. CRISPR, while powerful, does not have a 100% success rate. However, you need 100% certainty in your product. Thats where the absolute quantification from ddPCR technology comes in. Here we explore several instances where ddPCR assays allow scientists to expand cell and gene therapy boundaries.

The ideal quality control assay for gene therapy would simultaneously quantify the transfer of genetic material and changes in expression. In their 2021 publication, Clarner et al. did just that using a one-step RT-ddPCR method. Their method quantified transgene expression and potency with both RNAi and augmentation vectors in vitroand in vivo,using non-human primate models. They noted that the absolute quantification from ddPCR reduced variability and provided a more streamlined workflow.

In a second example, CRISPR can generate knock-ins to increase gene dosage, which is technically challenging because integration efficiency is low. The process of developing knock-ins becomes time and resource-intensive and requires processing many failures.CRISPR SNIPERuses ddPCR technology to quantify knock-ins with precision and efficiency. Because ddPCR assays partition donor DNA into 20,000 droplets, CRISPR SNIPER can accurately measure low-frequency integration events that qRT-PCR simply cannot detect.

Finally, ddPCR methods are excellent for cell therapy transgene quantification. CAR-T cells are generated using viral vectors that insert transgenes into T-cell DNA. However, the number of integrated transgene copies can be variable.Lu et al. used ddPCR technology to accurately measure transgene copy insertion in CAR-T cells with high levels of reproducibility.

Across the board, ddPCR assays are uniquely positioned to catapult cell and gene therapies to the next level, standardizing and safeguarding the production process worldwide. ddPCR methods possess the accuracy, precision, and reproducibility required for cell and gene therapy quality control. Across applications from knock-in generation to CAR-T cell transgene quantification, this technology allows scientists to focus on what they do best: science. Meanwhile, ddPCR technology can take care of quality control.

Learn more about ddPCR applications for cell and gene therapy manufacturing.

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Next-level quality control in cell and gene therapy - BioPharma Dive

1. CRISPR 'cousin' put to the test in landmark heart-disease trial  Nature.com
2. CRISPR cure for high cholesterol enters first human trial  Freethink
3. Boston biotech Verve tests 'CRISPR 2.0 in a patient for the first time  The Boston Globe
4. Edits to a cholesterol gene could stop the biggest killer on earth  MIT Technology Review
5. Verve starts first human test of gene editing treatment for heart disease  BioPharma Dive
6. View Full Coverage on Google News

Original post:
CRISPR 'cousin' put to the test in landmark heart-disease trial - Nature.com

Can CRISPR edit out a heart attack? What happens on #GutTok? And is health care recession-proof?

We cover all that and more this week on The Readout LOUD, STATs biotech podcast. Sek Kathiresan, cardiologist and CEO of Verve Therapeutics, joins us to explain the companys work on preventing heart disease with genome editing. Then, STATs Isabella Cueto joins us to discuss Hot girls have IBS, an internet in-joke that evolved into a movement for people with chronic illness. We also break down the latest news in the life sciences, including a long-awaited victory for Novavax and ostensible good news for biotech.

For more on what we cover, heres the latest on Verve; heres the story on hot girls have IBS; heres the news on Novavax;heres where you can find episodes of Color Code; heres where you can subscribe to the First Opinion Podcast;and heres our complete coverage of the Covid-19 pandemic.

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Be sure to sign up on Apple Podcasts, Spotify, Stitcher,Google Play, or wherever you get your podcasts.

And if you have any feedback for us topics to cover, guests to invite, vocal tics to cease you can email [emailprotected].

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CRISPR for the heart, biotech's recovery, & what it means to be a 'hot girl' - STAT

BOSTON Researchers from the Center for Regenerative Medicine at Boston Medical Center and Boston University School of Medicine used variants of CRISPR to understand the functions of the genes that cause emphysema and chronic obstructive pulmonary disease (COPD). Published in Science Advances, researchers discovered functional consequences by turning off the expression of the gene that contributes to the pathogenesis of these diseases.

This is the first time that CRISPRi and CRISPRa have been applied in human induced pluripotent stem cells to understand the functional role of these genes, says Andrew Wilson, MD, a pulmonologist at Boston Medical Center and associate professor of medicine at Boston University School of Medicine. It gets us closer to understanding how inherited factors help contribute to susceptibility to emphysema.

COPD and emphysema is the third leading cause of death worldwide, creating a significant burden of disease. Emphysema is a complex genetic disease caused by a mutation or variant in a number of genes that contribute to making some individuals more susceptible to disease than others. Genome-wide association studies (GWAS) have implicated variants in or near a growing number of genes, but understanding their functions and how they potentially contribute to the development of COPD and emphysema is quite limited.

There have been no new significant pharmacological agents developed to help treat the large number of patients afflicted with COPD or emphysema worldwide, says Rhiannon Werder, MD, a postdoctoral fellow at the Center for Regenerative Medicine at Boston Medical Center and Boston University School of Medicine. Our hope is that this study will help in the understanding of the genetics of the disease, improve our understanding of how the disease occurs at a cellular level, and support the development of new therapies to treat these conditions.

Researchers devised a system using variants of CRISPR to either turn off expression of a gene of interest using CRISPR interference (CRISPRi) or overexpress a gene of interest using CRISPR activation (CRISPRa) in induced pluripotent stem cells (iPSCs). Researchers grew these cells in a dish and differentiated them to generate cells that reside in the lung. The cell type studied is called the type 2 alveolar epithelial cell, a progenitor cell for the alveolus the alveolus is the part of the lung where gas exchange occurs and is the structure that is damaged in emphysema. So by understanding how GWAS genes affect type 2 cells, researchers can start to understand how these might contribute to diseases that affect these cells, like emphysema.

Once type 2 cells were generated, researchers then used CRISPRi to turn off expression of nine different GWAS genes and analyzed them to see how the cells were affected, especially their ability to proliferate, something that they need to be able to do in response to injury like that which occurs in emphysema. Researchers noticed that turning off one particular gene, desmoplakin (DSP), caused the cells to increase their proliferation and increased their expression of genes associated with cellular maturation. Researchers found that cells in which DSP expression was turned off before smoke exposure turned off expression of cell junction genes to a greater degree than in controls. These were also better at forming new colonies, a measure of progenitor function, than controls. Researchers then looked in mice that had DSP deleted from their lung epithelial cells, compared to control mice with normal DSP. Researchers found that the type 2 cells in the DSP deletion mice were more proliferative following injury, consistent with findings in human iPSC-derived type 2 cells.

DSP appears to modulate the proliferative capacity of type 2 cells at baseline and following injuries that are relevant to human disease, such as smoke exposure. Lower levels of DSP expression increase the proliferative capacity of type 2 cells in the system, potentially making them better able to respond to an injury. In contrast, higher levels of expression as found in cells containing the variant associated with COPD risk by GWAS appear to make the cells less proliferative after smoke exposure, potentially explaining how this gene contributes to disease.

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CRISPR Technology Highlights Genes That Contribute to the Development of Emphysema and COPD - Boston Medical Center

Kenneth Research

Key Companies Covered in the Global Gene Therapy Market by Kenneth Research are Kineta, Inc., Novartis AG, Amgen Inc., bluebird bio, Inc., Gilead Sciences, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

New York, July 13, 2022 (GLOBE NEWSWIRE) -- According to the World Health Organization (WHO), around 10 million deaths, or nearly 1 in 6 deaths, were caused by cancer in 2020, making it the top cause of death globally. Breast, lung, colon, rectum, and prostate cancers are the most prevalent types of cancer. If found early and appropriately treated, many tumors (30% to 50%) are curable. According to the American Cancer Society (ACS), 1,918,030 new cancer cases and 609,360 cancer deaths are expected in 2022, with lung cancer as the primary cause of death accounting for about 350 of those fatalities daily in the United States.

In recent research titled Global Gene Therapy Market, Kenneth Research provided a brief overview of market elements including growth drivers, restraint factors, current market trends, and potential for future growth. The influence of COVID-19 and its effects on end-users are both thoroughly examined in the market research report, which covers the forecast period, i.e., 2022-2031. In addition, the research study examines the product portfolios and market expansion plans of the principal competitors.In 2020, according to the World Cancer Research Fund (WCRF), there were 18 million new cases of cancer worldwide. 9.3 million of these instances involved men, while 8.8 million involved women. The growth of the global gene therapy market can be attributed on account of the rising prevalence of cancer cases. Also, the adoption of gene therapies for the treatment of cancer is predicted to grow the market further. For instance, at the University of Pennsylvania, the first trial for testing a CRISPR-created cancer medicine was launched in the United States in 2019.CRISPR is a gene-editing-tool, that can modify any DNA segment within the 3 billion letters of the human genome. The global gene therapy market is expected to gather around USD 6 billion in revenue by 2031 and grow with a CAGR of ~34% over the forecast period. Get A Sample Copy of This Report @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented on the basis of region into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. On the back of rapid rising cancer incidence rates, and the availability of high disposable income, the market in North America is predicted to experience significant expansion over the course of the forecast period. For instance, the Cancer Facts & Figures 2021 by the American Cancer Society, the study estimates that 1.9 million new instances of cancer were diagnosed and 608,570 cancer deaths in the United States in 2021. Also, an increase in the demand for gene-therapy-related R&D activities further helps the growth of the market. According to the World Bank Data, the domestic general government healthcare expenditure in the U.S. was 5,552.60 IN 2019 whereas in Canada the domestic general health care expenditure was 3,873.70 in 2019. Thus, a rise in government health care support is expected to expand the gene therapy-related R&D activities and further aid on to improve the market in the region.On the other hand, the global gene therapy market in the Asia Pacific region is anticipated to experience the greatest CAGR throughout the forecast period owing to the growing population in the region and increased approval and availability of gene therapy products. According to the World Bank data, the total population of China was 1.41 billion in 2020 whereas, India had 1.38 billion people in 2020. As the population grows, the likelihood of contracting a disease increases. Additionally, it is anticipated that increased government efforts to upgrade the health care infrastructure and rising healthcare costs in that region are expected to expand the industry. Also, the health care expenditure in Japan in 2019 was 10.74% whereas, in China, the GDP was 5.35%. In addition to that, the domestic general government health expenditure per capita for Japan was 3,846.54 in 2019 and China was 492.72 in 2019. Thus, growing health care expenditure and government support in health care expansion are further expected for the growth of the market in the region.

Browse to access In-depth research report on Gene Therapy Market with detailed charts and figures: https://www.kennethresearch.com/report-details/gene-therapy-market/10070542

The study further incorporates Y-O-Y Growth, demand & supply and forecasts future opportunities in North America (U.S., Canada), Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC[Finland, Sweden, Norway, Denmark], Poland, Turkey, Russia, Rest of Europe), Latin America (Brazil, Mexico, Argentina, Rest of Latin America), Asia Pacific(China, India, Japan, South Korea, Indonesia, Singapore, Malaysia, Australia, New Zealand, Rest of Asia Pacific), Middle East and Africa(Israel, GCC[Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of the Middle East and Africa).The global gene therapy market is segmented by indication into cancer, metabolic disorders, eye disorders, cardiovascular diseases, and others. Among that the cancer segment is predicted to hold the largest share over the forecast period. On account of the growing widespread presence of cancer cases, the growth of the market can be accredited. The estimated number of new cases of cancer patients in India was around 11,57,294 cases which had risen to 13,24,413 total cases in 2020. In addition to that, the total number of cancer patients was 1,708,921 in 2018 in the U.S., according to the Centers for Disease Control and Prevention (CDC) which got increased to an estimated rate of 1.8 million new cases in 2020. The statistical studies exhibit an increasingly widespread of the disease worldwide which is expected to drive the growth of the segment. Gene therapies are used to treat a variety of malignancies, including those of the brain, lung, breast, pancreatic, liver, prostate, bladder, head & neck, skin, and ovary. For instance, according to the World Cancer Research Fund (WCRF), the most common cancers around the world were breast and lung cancers, accounting to 12.5% and 12.2% respectively of all new cases that were expected to be diagnosed in 2020. Also, there were 1.9 million new instances of colorectal cancer, accounting for 10.7% of all cancer cases in 2020.

Get a Sample PDF of Global Gene Therapy Market @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented by end-user into pharma & biotech, and academia. Numerous ongoing researches and studies have been conducted in the pharma and biotech sector which is anticipated to account for the growth of the segment. For instance, based on a study by PhRMA, there were 289 gene therapies done in clinical development by biopharmaceutical companies in 2018 which had increased to 362 gene therapies in 2020. Also, 6 diseases were already being treated using gene therapy, whereas 362 cell and gene therapies were in the development stage in 2020. In addition to that, 9 cell or gene therapy products have been approved by U.S. Food and Drug Administration (FDA) as of February 2020; they are used to treat cancer, eye conditions, and uncommon inherited diseases.

The global gene therapy market is also segmented on the basis of technology and application.

Global Gene Therapy Market, Segmentation by Technology:

Global Gene Therapy Market, Segmentation by Application:

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Some of the well-known leaders in the global gene therapy market that are included in our report are Kineta, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

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Anticholinergic Drugs Market Segmentation by Product Type (Natural, Semi-Synthetic Compound, and Synthetic Compound); by Route of Administration (Parenteral, Topical, and Oral); by Application (Muscle Spasm, Parkinsons Disease, Irritable Bowel Syndrome, Chronic Obstructive Pulmonary Disease, and Overactive Bladder); and by Channel of Distribution (Hospitals, Retail Pharmacies, and Online Pharmacies)-Global Demand Analysis & Opportunity Outlook 2031

Global Vaginal Slings Market Segmentation by End-Use (Clinics, Hospitals, and Ambulatory Surgical Centers); and by Slings Type (Conventional, and Advanced Vaginal Slings)-Demand Analysis & Opportunity Outlook 2031

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Here is the original post:
Global Gene Therapy Market to Garner a Revenue of About USD 6 Billion by 2031 by Growing with a CAGR of ~34% During 2022-2031; Market to Grow on...

What happened

The downtrodden biotech space has kicked off the second half of 2022 with a boom. Hard-hit gene-editing and gene therapy companies in particular have started the back half of the year on the right foot. Underscoring this point, Bluebird Bio (BLUE 6.97%) stock has already risen by 17% over the holiday-shortened week as of Thursday's closing bell, according to data provided by S&P Global Market Intelligence.

What's more, shares of CRISPR Therapeutics (CRSP 0.21%) have gained 22.6% over the same period, and fellow gene editor Editas Medicine (EDIT -0.20%) also saw its equity rise in price by a healthy 20.7% this week. By contrast, Bluebird and Editas shares both fell by over 50% over the first six months of 2022, while CRISPR's stock price stumbled by a noteworthy 20% during the first half of the year.

Image Source: Getty Images.

What's behind this sudden trend reversal? The most likely explanation is simply short-sellers covering their positions (buying back their borrowed shares). In keeping with this theme, Bluebird, Editas, and CRISPR all saw a sharp rise in their short interest during the first six months of 2022. Short-sellers piled into these three names earlier this year due to the fact that they are all cash flow negative, which is a tough spot to be in during a persistent bear market and an era of rising interest rates. Bluebird, in fact, is staring down a serious cash crunch at the moment.

Short-sellers, for their part, are probably backing away at this stage for no other reason than to play it safe in the event that big pharma starts to go bargain shopping.

Why might big pharma target beaten-down gene-editing and gene therapy companies in the second half of the year? The key reason is that these high-value fields are starting to move beyond the research stage of their life cycle and into the realm of commercially available therapies.

Speaking to this point, Bluebird's gene therapies for beta thalassemia and cerebral adrenoleukodystrophy appear to be on their way toward a formal approval from the Food and Drug Administration (FDA) following a pair of positive advisory committee votes last month. What's more, CRISPR is also expected to file for regulatory approval for its Vertex Pharmaceuticalspartnered blood disorder candidate, exa-cel, later this year.

Are any of these three biotech stocks still worth buying? CRISPR is arguably the most attractive bargain among the three. The company's ex-vivo gene-editing platform has posted stellar trial results so far, and Vertex could very well decide to buy its partner as a result.

Bluebird, on the other hand, is a tough call. The company ought to have a compelling buyout case if the FDA does grant it a pair of approvals soon. The bad news is that the biotech's balance sheet may force a sale at a heavily discounted price (relative to the commercial potential of its lead assets).

Finally, Editas might simply get lost in the mix when everything is said and done. There are several gene-editing companies vying for the spot of top dog, and Editas' clinical pipeline lags in several key areas at the moment. Time will tell.

George Budwell has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics, Editas Medicine, and Vertex Pharmaceuticals. The Motley Fool recommends Bluebird Bio. The Motley Fool has a disclosure policy.

See more here:
Why Shares of Bluebird Bio, CRISPR Therapeutics, and Editas Medicine Soared This Week - The Motley Fool

Can gene-editing technology CRISPR create new crops that help fight climate change as they grow? Thats what a group of researchers hopes to do with $11 million in funding from the Chan Zuckerberg Initiative. The funding will go toward efforts to enhance plants starting with rice and soil so that theyre better at trapping carbon dioxide. The effort, which was announcedlast week, is being led by the Innovative Genomics Institute, which was founded byNobel laureateand co-inventor of CRISPR Jennifer Doudna.

[Jennifer] and I saw eye to eye on climate and how big of a problem it is in the world. And we just didnt want to sit on the sidelines anymore, says Innovative Genomics Institute (IGI) executive director Brad Ringeisen.

Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.

The rice genome is easier to manipulate than other crops, according to Ringeisen, in part because its already been studied a lot and iswell understood. One of the scientists involved in IGIs initiative is Pamela Ronald, whose research is widely known for leading to thedevelopment of rice varietiesthat tolerate flooding for much longer than other types using a different type of genetic engineering thats more likeprecision breeding. That rice is now grown by more than 6 million farmers across India and Bangladesh,according toRonalds laboratory at the University of California, Davis.

Read the original:
Climate change-fighting rice? Plants trap carbon dioxide as they grow and CRISPR gene editing can optimize this process - Genetic Literacy Project

In a trio of studies published on June 27 in the journalNature Microbiology, researchers at The University of Texas at Austin have discovered "fingerprints" of mysterious viruses hidden in an ancient group of microbes that may include the ancestors of all complex life on Earth: from fungi to plants to humans.

Ths discovery is significant; it explores the hypothesis that viruses were imperative to the evolution of humans and other complex life forms.

These microbes known as Asgard archaea after the abode of the gods in Norse mythology are usually found in the frigid sediments deep in the ocean and in boiling springs, and existed on Earth before the firsteukaryoticcells, which carry theirDNAinside a nucleus.

Some scientists have hypothesized that viruses may have played in role in how life forms first came to be by infecting the Asgard archaea. They may have even given rise to some of the first precursors to the nucleus. But, no Asgard-infecting viruses had been discovered hitherto. The latest research by Ian Rambo (a former doctoral student at UT Austin) and other members of Brett Baker's lab sheds light on how viruses might have played a role in this billions-year-old history.

"These are the first studies investigating Asgard archaeal viruses; there was nothing known before," Susanne Erdmann, group leader of the archaeal virology research group at the Max Planck Institute for Marine Microbiology in Bremen, Germany, who was not involved in the studies, toldLive Science. In the future, this line of research may reveal if and how viruses were involved in the emergence of eukaryotic cells on Earth, she said.

In the new research, scientists searched for evidence of viral infection embedded in the DNA of Asgard archaea - which comes in the form of viral DNA called "CRISPR spacers."

According to Rambo, most people who think of CRISPR relate it to the famous gene-editing tool that allows scientists to easily manipulate genetic sequences. This tool was originally adapted from the natural defense mechanisms of bacteria and archaea.

CRISPR refers to a region of DNA made up of short, repeated sequences with "spacers" sandwiched between each repeat. Interestingly, bacteria and archaea swipe these spacers from viruses that infect them, and the cells maintain a memory bank of viral DNA that helps them recognize the viruses should they attack again.

"It's an adaptive immune system that remembers these previous infections," said Rambo, who is now a postdoctoral scholar with the USDA's Agricultural Research Service.

Rambo and his colleagues had hunted in the Guaymas Basin in the Gulf of California the body of water between Baja California and mainland Mexico for such DNA spacers in Asgard archaea specimens collected from sediments near hydrothermal vents, roughly 1.25 miles (2 kilometers) beneath the water's surface.

The team matched the spacers they found to longer stretches of viral DNA gathered from the deep-sea environment.

The researchers could infer the kinds of proteins the various genes code for and how the viruses might function, working with viral DNA.

But, eventually, they could only figure out the functions of some of the viruses' genes; the functions of the vast majority of the genes are still unknown, Erdmann said. Also, because CRISPR doesn't work against all viruses, many more Asgard-infecting viruses are yet to be discovered, she said.

These hidden viruses could be found by growing Asgard archaea in the lab. "However, culturing Asgard archaea has been proven very difficult," Erdmann said. Until now, only one research group has managed to culture Asgard archaea, and it took them 12 long years to do it as archaeal cells take weeks to replicate.

But until more Asgards can be grown in the lab, CRISPR spacer matching is probably the most efficient way to find more viruses, Krupovic said. As more viruses are found, their role in the emergence of eukaryotes, including humans, may gain more clarity, added Rambo.

Read the original post:
Newly discovered viruses can offer clues about the rise of complex life on Earth - Interesting Engineering

Smartphones, superglue, electric cars, video chat. When does the wonder of a new technology wear off? When you get so used to its presence that you dont think of it anymore? When something newer and better comes along? When you forget how things were before?

Whatever the answer, the gene-editing technology CRISPR has not reached that point yet. Ten years after Jennifer Doudna and Emmanuelle Charpentier first introduced their discovery of CRISPR, it has remained at the center of ambitious scientific projects and complicated ethical discussions. It continues to create new avenues for exploration and reinvigorate old studies. Biochemists use it, and so do other scientists: entomologists, cardiologists, oncologists, zoologists, botanists.

Cathie Martin, a botanist at the John Innes Centre in Norwich, England, and Charles Xavier, founder of the X-Men superhero team: They both love mutants.

But while Professor X has an affinity for superpowered human mutants, Dr. Martin is partial to the red and juicy type. We always craved mutants, because that allowed us to understand functionality, Dr. Martin said of her research, which focuses on plant genomes in the hopes of finding ways to make foods especially tomatoes in her case healthier, more robust and longer lasting.

When CRISPR-Cas9 came along, one of Dr. Martins colleagues offered to make her a mutant tomato as a gift. She was somewhat skeptical, but, she told him, I would quite like a tomato that produces no chlorogenic acid, a substance thought to have health benefits; tomatoes without it had not been found before. Dr. Martin wanted to remove what she believed was the key gene sequence and see what happened. Soon a tomato without chlorogenic acid was in her lab.

Instead of looking for mutants, it was now possible to create them. Getting those mutants, it was so efficient, and it was so wonderful, because it gave us confirmation of all these hypotheses we had, Dr. Martin said.

Most recently, researchers at Dr. Martins lab used CRISPR to create a tomato plant that can accumulate vitamin D when exposed to sunlight. Just one gram of the leaves contained 60 times the recommended daily value for adults.

Dr. Martin explained that CRISPR could be used across a broad spectrum of food modifications. It could potentially remove allergens from nuts and create plants that use water more efficiently.

I dont claim that what we did with vitamin D will solve any food insecurity problems, Dr. Martin said, but its just a good example. People like to have something that they can hang on to, and this is there. Its not a promise.

Infectious Disease

Christian Happi, a biologist who directs the African Centre of Excellence for Genomics of Infectious Diseases in Nigeria, has spent his career developing methods to detect and contain the spread of infectious diseases that spread to humans from animals. Many of the existing ways to do so are costly and inaccurate.

For instance, in order to perform a polymerase chain reaction, or PCR, test, you need to go extract RNA, have a machine thats $60,000 and hire someone who is specially trained, Dr. Happi said. Its both costly and logistically implausible to take this kind of testing to most remote villages.

Recently, Dr. Happi and his collaborators used CRISPR-Cas13a technology (a close relative of CRISPR-Cas9) to detect diseases in the body by targeting genetic sequences associated with pathogens. They were able to sequence the SARS-CoV-2 virus within a couple of weeks of the pandemic arriving in Nigeria and develop a test that required no on-site equipment or trained technicians just a tube for spit.

If youre talking about the future of pandemic preparedness, thats what youre talking about, Dr. Happi said. Id want my grandmother to use this in her village.

The CRISPR-based diagnostic test functions well in the heat, is quite easy to use and costs one-tenth of a standard PCR test. Still, Dr. Happis lab is continually assessing the accuracy of the technology and trying to persuade leaders in the African public health systems to embrace it.

He called their proposal one that is cheaper, faster, that doesnt require equipment and can be pushed into the remotest corners of the continent. This would allow Africa to occupy what I call its natural space.

Hereditary Illness

In the beginning there was zinc finger nuclease.

That was the gene-editing tool that Gang Bao, a biochemical engineer at Rice University, first used to try to treat sickle cell disease, an inherited disorder marked by misshapen red blood cells. It took Dr. Baos lab more than two years of development, and then zinc finger nuclease would successfully cut the sickle cell sequence only around 10 percent of the time.

Another technique took another two years and was only slightly more effective. And then, in 2013, soon after CRISPR was used to successfully edit genes in living cells, Dr. Baos team changed tack again.

From the beginning to having some initial results, CRISPR took us like a month, Dr. Bao said. The method successfully cut the target sequence around 60 percent of the time. It was easier to make and more effective. It was just amazing, he said.

The next challenge was to determine the side effects of the process. That is, how did CRISPR affect genes that werent being purposefully targeted? After a series of experiments in animals, Dr. Bao was convinced that the method would work for humans. In 2020 the Food and Drug Administration approved a clinical trial, led by Dr. Matthew Porteus and his lab at Stanford University, that is ongoing. And there is also hope that with CRISPRs versatility, it might be used to treat other hereditary diseases. At the same time, other treatments that have not relied on gene editing have had success for sickle cell.

Dr. Bao and his lab are still trying to determine all the secondary and tertiary effects of using CRISPR. But Dr. Bao is optimistic that a safe and effective gene-editing treatment for sickle cell will be available soon. How soon? I think another three to five years, he said, smiling.

Cardiology

It is hard to change someones heart. And thats not just because we are often stubborn and stuck in our ways. The heart generates new cells at a much slower rate than many other organs. Treatments that are effective in other parts of the human anatomy are much more challenging with the heart.

It is also hard to know what is in someones heart. Even when you sequence an entire genome, there are often a number of segments that remain mysterious to scientists and doctors (called variants of uncertain significance). A patient might have a heart condition, but theres no way to tie it definitively back to their genes. You are stuck, said Dr. Joseph Wu, director of the Stanford Cardiovascular Institute. So traditionally we would just wait and tell the patient we dont know whats going on.

But over the past couple of years, Dr. Wu has been using CRISPR to see what kind of effects the presence and absence of these befuddling sequences have on heart cells, simulated in his lab with induced pluripotent stem cells generated from the blood. By cutting out particular genes and observing the effects, Dr. Wu and his collaborators have been able to draw links between the DNA of individual patients and heart disease.

It will be a long time before these diseases can be treated with CRISPR, but diagnosis is a first step. I think this is going to have a big impact in terms of personalized medicine, said Dr. Wu, who mentioned that he found at least three variants of uncertain significance when he got his own genome sequenced. What do these variants mean for me?

Sorghum is used in bread, alcohol and cereal all over the world. But it hasnt been commercially engineered to the same degree as wheat or corn, and, when processed, it often isnt as tasty.

Karen Massel, a biotechnologist at the University of Queensland in Australia, saw quite a bit of room for improvement when she first started studying the plant in 2015. And because millions of people eat sorghum worldwide, if you make a small change you can have a huge impact, she said.

She and her colleagues have used CRISPR to try to make sorghum frost tolerant, to make it heat tolerant, to lengthen its growth period, to change its root structure we use gene editing across the board, she said.

Not only could this lead to more delicious and healthier cereal, but it could also make the plants more resistant to the changing climate, she said. But it is still no small task to accurately edit the genomes of crops with CRISPR.

Half the genes that we knock out, we just have no idea what they do, Dr. Massel said. The second we try to get in there and play God, we realize were a bit out of our depth. But, using CRISPR combined with more traditional breeding techniques, Dr. Massel is optimistic, despite being a self-described pessimist. And she hopes that further advances will lead to commercializing gene-edited foods, making them more accessible and more acceptable.

In 2012, a 6-year-old girl was suffering from acute lymphoblastic leukemia. Chemotherapy had been unsuccessful, and the case was too advanced for a bone-marrow transplant. There didnt seem to be any other options, and the girls physicians told her parents to go back home.

Instead, they went to the Childrens Hospital of Philadelphia, where doctors used an experimental treatment called chimeric antigen receptor (CAR) T-cell therapy to turn the girls white blood cells against the cancer. Ten years later, the girl is cancer free.

Since then, Dr. Carl June, a medical professor at the University of Pennsylvania who helped develop CAR T-cell therapy, and his collaborators, including Dr. Ed Stadtmauer, a hematologist-oncologist at Penn Medicine, have been working to improve it. That includes using CRISPR, which is the simplest and most accurate tool to edit T-cells outside the body. Dr. Stadtmauer, who specializes in dealing with various types of blood and lymph system cancers, said that the last decade or so has just seen a revolution of treatment of these diseases; its been rewarding and exciting.

Over the past couple of years, Dr. Stadtmauer helped run a clinical trial in which T-cells that underwent significant CRISPR editing were inserted into patients with treatment-resistant cancers. The results were promising.

Nine months into the trial the edited T-cells had not been rejected by the patients immune systems and were still present in the blood. The real benefit is that scientists now know that CRISPR-aided treatments are possible.

Even though its really sort of science fiction-y biochemistry and science, the reality is that the field has moved tremendously, Dr. Stadtmauer said. He added that he was less excited by the science than how useful CRISPR had become. Every day I see maybe 15 patients who need me, he said. Thats what motivates me.

More:
The Many Uses of CRISPR: Scientists Tell All - The New York Times

Over the last several decades, genetic research has seen incredible advances in gene sequencing technologies. In 2004, scientists completed the Human Genome Project, an ambitious project to sequence the human genome, which cost $3 billion and took 10 years. Now, a person can get their genome sequenced for less than $1,000 and within about 24 hours.

Scientists capitalized on these advances by sequencing everything from the elusive giant squid to the Ethiopian eggplant. With this technology came promises of miraculous breakthroughs: all diseases would be cured and world hunger would be a thing of the past.

So, where are these miracles?

We need about 60 to 70% more food production by 2050.

In 2015, a group of researchers founded Yield10 Bioscience, an agriculture biotech company that aimed to use artificial intelligence to start making those promises into reality.

Two things drove the development of Yield10 Bioscience.

One, obviously, [the need for] global food security: we need about 60 to 70% more food production by 2050, explained Dr. Oliver Peoples, CEO of Yield10 Bioscience, in an interview with Freethink. And then, of course, CRISPR.

It turns out that having the tools to sequence DNA is only step one of manufacturing the miracles we were promised.

The second step is figuring out what a sequence of DNA actually does. In other words, its one thing to discover a gene, and it is another thing entirely to discover a genes role in a specific organism.

In order to do this, scientists manipulate the gene: delete it from an organism and see what functions are lost, or add it to an organism and see what is gained. During the early genetics revolution, although scientists had tools to easily and accurately sequence DNA, their tools to manipulate DNA were labor-intensive and cumbersome.

Its one thing to discover a gene, and it is another thing entirely to discover a genes role in a specific organism.

Around 2012, CRISPR technology burst onto the scene, and it changed everything. Scientists had been investigating CRISPR a system that evolved in bacteria to fight off viruses since the 80s, but it took 30 years for them to finally understand how they could use it to edit genes in any organism.

Suddenly, scientists had a powerful tool that could easily manipulate genomes. Equipped with DNA sequencing and editing tools, scientists could complete studies that once took years or even decades in mere months.

Promises of miracles poured back in, with renewed vigor: CRISPR would eliminate genetic disorders and feed the world! But of course, there is yet another step: figuring out which genes to edit.

Over the last couple of decades, researchers have compiled databases of millions of genes. For example, GenBank, the National Institute of Healths (NIH) genetic sequence database, contains 38,086,233 genes, of which only tens of thousands have some functional information.

For example, ARGOS is a gene involved in plant growth. Consequently, it is a very well-studied gene. Scientists found that genetically engineering Arabidopsis, a fast-growing plant commonly used to study plant biology, to express lots of ARGOS made the plant grow faster.

Dozens of other plants have ARGOS (or at least genes very similar to it), such as pineapple, radish, and winter squash. Those plants, however, are hard to genetically manipulate compared to Arabidopsis. Thus, ARGOSs function in crops in general hasnt been as well studied.

The big crop companies are struggling to figure out what to do with CRISPR.

CRISPR suddenly changed the landscape for small groups of researchers hoping to innovate in agriculture. It was an affordable technology that anyone could use but no one knew what to do with it. Even the largest research corporations in the world dont have the resources to test all the genes that have been identified.

I think if you talk to all the big crop companies, theyve all got big investments in CRISPR. And I think theyre all struggling with the same question, which is, This is a great tool. What do I do with it? said Dr. Peoples.

The algorithm can identify genes that act at a fundamental level in crop metabolism.

The holy grail of crop science, according to Dr. Peoples, would be a tool that could identify three or four genetic changes that would double crop production for whatever youre growing.

With CRISPR, those changes could be made right now. However, there needs to be a way to identify those changes, and that information is buried in the massive databases.

To develop the tool that can dig them out, Dr. Peoples team merged artificial intelligence with synthetic biology, a field of science that involves redesigning organisms to have useful new abilities, such as increasing crop yield or bioplastic production.

This union created Gene Ranking Artificial Intelligence Network (GRAIN), an algorithm that evaluates scientific databases like GenBank and identifies genes that act at a fundamental level in crop metabolism.

That fundamental level aspect is one of the keys to GRAINs long-term success. It identifies genes that are common across multiple crop types, so when a powerful gene is identified, it can be used across multiple crop types.

For example, using the GRAIN platform, Dr. Peoples and his team identified four genes that may significantly impact seed oil content in Camelina, a plant similar to rapeseed (true canola oil). When the researchers increased the activity of just one of those genes via CRISPR, the plants had a 10% increase in seed oil content.

Its not quite a miracle yet, but with more advances in gene editing and AI happening all the time, the promises of the genetic revolution are finally starting to pay off.

Wed love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us attips@freethink.com.

Continued here:
How artificial intelligence is boosting crop yield to feed the world - Freethink

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Highlights of TOC: Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market

1 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Overview

2 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Competitions by Manufacturers

3 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Capacity, Production, Revenue (Value) by Region (2022-2029

4 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Supply (Production), Consumption, Export, Import by Region (2022-2029)

5 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Production, Revenue (Value), Price Trend by Type

6 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Analysis by Application

7 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Manufacturers Profiles/Analysis

8 Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Manufacturing Cost Analysis

9 Industrial Chain, Sourcing Strategy and Downstream Buyers

10 Marketing Strategy Analysis, Distributors/Traders

11 Market Effect Factors Analysis

12 Global Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Forecast (2022-2029)

13 Research Findings and Conclusion

14 Appendix

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Goals and objectives of the Global Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Market Study

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Asia-Pacific Health Screening Market Size, Top Leading Countries, Companies, Consumption, Drivers, Trends, Revenue, Regional Analysis, Challenges

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Clustered Regularly Interspersed Short Palindromic Repeats (CRISPR) Market Size, Top Leading Countries, Companies, Consumption, Drivers, Trends,...

Blaupunkt, a German audio electronics brand has launched a new set of TWS earbuds in India called theBTW100TWS. They come withENC CRISPR Technologythat filters ambient noises while on a call and only picks up human voices. The earbuds are budget-friendly and have great German quality and technology. These TWS earbuds come with a stem design and charge inside an oval shaped charging case. Moreover, they have a battery life of up to 40 hours playtime.

The earbuds are powered by a powerful 10mm driver that produces punchy bass along with crystal clear mids and highs. The speakers produce Stereo high definition sound. For connectivity, the earbuds use Bluetooth 5.1 which enables a maximum range of 30ft without signal loss or mic dropout. These earbuds also feature an 80ms low latency mode for gaming and are enabled with Intuitive Touch Controls.

The Blaupunkt BTW100 TWS have a straight stem with chrome edges while the charging case has a clamshell-like design. The charging case packs a large 400mAh battery that is backed by TurboVolt Charging feature providing 1 hour of playback time with 15 minutes. Additional features include sweat, water & dust resistance.

The Blaupunkt BTW100 TWS earbuds are priced atRs 2,999and are available in two colour options Black and White. The product is available onAmazon.

Link:
Blaupunkt Launched Its BTW100 Truly Wireless Bluetooth Earbuds with ENC CRISPR Technology - Digit

Larger, deeper root systems can help store more carbon in the soil, because if a plant dies and parts of it are deep underground, the carbon in those pieces is less likely to make its way back into the air quickly. Roots arent the only possible storage option, Ringeisen says. Modified plants could also be used to make bio-oil or biochar, which can be pumped deep underground for storage.

Optimizing plants for carbon removal will be challenging, says Daniel Voytas, a genetic engineer at the University of Minnesota and a member of IGIs scientific advisory board.

Many of the traits that researchers want to alter in plants are influenced by multiple genes, which can make precise editing difficult, he says. And while some plants, like tobacco and rice, have been so extensively studied that researchers broadly understand how to tweak them, the genetics of others are less well understood.

Most of the IGIs initial research on photosynthesis and root systems will focus on rice, Ringeisen says. At the same time, the institute will also work on developing better gene-editing techniques for sorghum, a staple crop that has been particularly tough for researchers to crack. The team hopes to eventually understand and potentially alter soil microbes as well.

This is not easy, but were embracing the complexity, Ringeisen says. Ultimately, he hopes that when it comes to climate change, plants and microbes and agriculture can actually be part of the solution, rather than part of the problem.

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This CRISPR pioneer wants to capture more carbon with crops - MIT Technology Review

Shares of CRISPR Therapeutics fell more than 11% on Monday as investors reacted negatively to the endorsement of a rival beta-thalassemia gene therapy developed by bluebird bio.

Last week, the U.S. Food and Drug Administrations Cell, Tissue and Gene Therapies Advisory Committee unanimously supported bluebirds beti-cel, a one-time gene therapy for patients with transfusion-dependent beta-thalassemia, a rare, inherited blood disorder caused by a genetic defect in hemoglobin.

Beti-cel, also known as betibeglogene autotemcel, is marketed in Europe as Zynteglo. Late-stage clinical data showed that 89% of patients who could be evaluated achieved transfusion independence following treatment with beti-cel, and safety data has been positive. The FDA is expected to give its final verdict on beti-cel by Aug. 19.

One day after the advisory committee endorsed beti-cel for beta-thalassemia, CRISPR Therapeutics and its partner Vertex Pharmaceuticalsreleasedpositive data for their gene therapy candidate, exa-cel. Exa-cel is a CRISPR-Cas9-based gene editing therapy for both transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD).

Data shared by the companies showed that 42 of 44 patients with TDT who received exa-cel have remained transfusion free for up to 37.2 months. The two patients who were not transfusion free had 75% and 89% reductions in transfusion volume, the companies said.

In SCD, the data was also positive. All 31 patients with sickle cell disease that is characterized by recurrent vaso-occlusive crises (VOCs) were free of the events following treatment with exa-cel. Data showed the patients had a duration of up to 32.3 months, CRISPR and Vertex reported, which expanded their partnership in this space last year.

Carmen Bozie, head of global medicines development and medical affairs at Vertex, touted the data. Bozie noted that of the 75 patients treated with exa-cel, 33 have one year or more of follow-up after infusion with the gene therapy. The data demonstrate the potential of exa-cel as a one-time functional cure for patients with transfusion-dependent beta-thalassemia or severe sickle cell disease, she said in a statement.

While bluebirds beti-cel was largely free of serious adverse events, Vertex and CRISPR reported that two of the 44 TDT patients experienced an SAE. One of the patients experienced three serious events that were connected to exa-cel, as well as busulfan, which was administered along with the gene therapy. That patient experienced hemophagocytic lymphohistiocytosis (HLH), a life-threatening condition related to excessive immune response, as well as acute respiratory distress syndrome and headache. The other patient experienced idiopathic pneumonia syndrome that was considered related to both exa-cel and busulfan, the companies said.

Among the 31 patients with SCD, there were no SAEs considered related to exa-cel.

CRISPR and Vertex are not alone in chasing bluebird bio to market with a gene therapy for beta-thalassemia. Editas Medicine is also developing its own gene therapy for the debilitating disease.

Earlier this year, Editas wonRare Pediatric Disease designationfor its experimental beta-thalassemia gene therapy, EDIT-301. The therapeutic is designed to edit the HBG1/2 promoter to disrupt the binding site of BCL11a and ameliorate disease symptoms.

In May, EDIT-301 won Orphan Drug designation for the treatment of beta-thalassemia and SCD. Editas expects to initiate a Phase I/II study of EDIT-301 in patients with transfusion-dependent beta-thalassemia later this year.

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CRISPR Tx Shares Fall as bluebird's Gene Therapy Soars - BioSpace