Archive for the ‘Crispr’ Category

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

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

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

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

Mass Use of Flying Jetpacks

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

An AI Machine As Your Boss

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

Your CRISPR Children

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

Live Forever

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

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

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

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

About TCR2 Therapeutics

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

Forward-looking Statements

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

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

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

Investor and Media Contact:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

However, CRISPR-Cas9 is not without its drawbacks.

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

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

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

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

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

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

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

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

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

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

Here are some of the most recent findings:

Additional reporting by Alina Bradford, Live Science contributor.

Additional resources

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What Is CRISPR? | Live Science

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Earlier Is Better

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

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

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

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

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

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

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

Turning Genes On And Off

One potential treatment, though, is gene editing.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Find more information about recruitment for this study here.

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

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On The Frontlines Of Autism Research: North Carolina Professors Study Early Detection, Treatment - Here And Now

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Why Investors Rotating Towards Aphria (APHA), CRISPR Therapeutics AG (CRSP) - US Post News

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

these proteins prevent neutrophils from migrating into the inflamed join

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

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

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

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

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

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

The findings were published in PNAS.

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

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

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

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

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

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

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

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

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

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

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Trends and Drivers

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

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

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

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

Key Players Mentioned in the Report are:

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

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

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

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

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CRISPR & CRISPR-associated (Cas) Genes Market Growth Opportunities and Forecast to 2027 - Fusion Science Academy

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

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

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

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

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

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

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

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

On Thursday, Shares ofCRISPR Therapeutics AG, (NASDAQ: CRSP), inclined/declined 56.57% and closed at $-3.12 in the last trading session.

Arithmetic Moving Averages CRSP:

Simple Moving Average (SMA) is easy to calculate and SMA20 one is principally looking at prime trends. The 50-day moving average is more responsive to price changes than the 200-day moving Whereas long-term trend followers generally use SMA200 and most shareholders will look for a cross up or down this average to means if the stock is in a bullish or bearish trend. SMA20 is the last stop on the bus for (ST) short-term traders. The CRISPR Therapeutics AG having its distance from 20-days simple moving average is -7.77%, and its distance from 50-days simple moving average is -10.46%, while it has a distance of 16.42% from the 200-days simple moving average.

Working over theproductivity proportionsof business stock, the speculator will discover its ROE, ROA, ROI remaining at -2.60%, -2.00% and -40.70%, individually.

ATR remains at 2.50 while Beta component of the stock stands at 0.00. The beta component is used to check the eccentrics of the stock. The CRSP stock remained -5.02% unpredictable for the week and -16.88% for the month.

Market capitalization is only an extravagant proclaim for a bright idea: it is the market estimation of an organizations remarkable offers. These Amount and numbers are found by taking the postscript cost and increasing it by the all outnumber of offers remarkable. Understanding the market top isnt merely sign if you nearly putting legitimately in stocks. It is additionally helpful for common reserve speculators, the same number of assets will list the normal or middle showcase capitalization of its property. As the name recommends, this gives the centre-ground of the stores value speculations, filling financial specialists in as to whether the reserve, for the most part, puts resources into large, mid-or little top stocks.

CRISPR Therapeutics AGTarget:

The EPS of the company is strolling at -0.46. The companys Market capitalization is $3.39BBillion.

As stocks have aP/S,P/EandP/Bestimations of 15.96, 0.00 and 5.24 separately. Its P/Cash is esteemed at 5.39.

Development in profit per offer is everything. The healthy future development in profit per share (EPS) is an amazingly significant factor in recognizing an underestimated stock. The effect of income development is exponential. As time goes on, the cost of a stock will typically go up in lockstep with its income (accepting the P/E proportion is steady). Hence stocks with higher profit development should offer the most elevated capital increases. Whats more, doubling-up the growth more than doubles the capital gain, due to the compounding effect.

Volume & Average Volume Shares:

Volume of the CRISPR Therapeutics AG exchanged hands with 1028892 shares compared to its average daily volume of 1.19M shares. Total volume is the number of shares or deals that point towards the whole activity of a security or market for a same period.

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Stock Highlights: :CRISPR Therapeutics AG, (NASDAQ: CRSP) - News Align

Apex Market Research provides market research reports from more than four years. Here we have issued the research report on Global CRISPR And CRISPR-Associated Genes Market Market. The report shows the all leading market players profiles. The report represents the full market analysis of the CRISPR And CRISPR-Associated Genes market with SWOT analysis, fiscal status, present development, acquisitions, and mergers. The CRISPR And CRISPR-Associated Genes market report represents the major challenges and newer opportunities. In-depth the newer growth tactics influenced by the industry manufactures the shows the international competitive scale of this market sector. The report gives the closer views to the global vendors to understand the CRISPR And CRISPR-Associated Genes market trends and meanwhile, generate important tactical actions to boost their business. The report investigates industry growth and risk factors as well as keep updates regarding development task happening in the globe market.

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Major Industry Player Profiles That Included by CRISPR And CRISPR-Associated Genes Market Research Report:

Thermo Fisher ScientificEditas MedicineCaribou BiosciencesCRISPR therapeuticsIntellia therapeutics, Inc.CellectisHorizon Discovery PlcSigma AldrichPrecision BiosciencesGenscriptSangamo Biosciences Inc.Lonza Group LimitedIntegrated DNA TechnologiesNew England BiolabsOrigene Technologies

Detailed view of CRISPR And CRISPR-Associated Genes Market:

For staying consistent in businesses and new initiate in the market, it is very essential to have a complete structure of the market holder. While thinking about this factor, the analysts provide a detailed view of the competitive strategies and landscape accepted by the principal players. The major feature of the market covered in this CRISPR And CRISPR-Associated Genes market report focused on opportunities, restraints, obstructions, global and regional distribution, market driving factors, and growth limiting factors.

The CRISPR And CRISPR-Associated Genes market report provides detailed data to mentor industry players while forming important business decisions. To provide this the report has used different analytical tools and procedures. In an aggressive market landscape, the report concentrate on every players detailed profile along with their product details, capacity, price, revenue, gross and contact information. CRISPR And CRISPR-Associated Genes market report consumes the production, import and export forecast by type, applications, and region with uniquely generated graph by our research team.

Market Type,

Genome EditingGenetic engineeringGRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering

Market Application,

Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

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The report focuses on regional segmentation to assist clients to understand region-wise analysis of CRISPR And CRISPR-Associated Genes market report. The report includes the case study of the top producers and consumers, focuses on product capacity, production, value, consumption, market share and growth opportunity in these key regions, covering North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia etc.), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa.

Key Objectives of This Report:

To redeem complete information to entrepreneurs about future products and technologies to be introduced in the market.To deliver access to unique information about top players of the Automotive Tyre market.The report focuses on feature about long-term and short-term strategies adopted by major players of the market along with their key developments.The report provides a country-wise analysis of the market helps to understand the market more precisely.To offer demand and growth trends of the market and segregation into segments.

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Global CRISPR And CRISPR-Associated Genes Market Insights 2019 Thermo Fisher Scientific, Editas Medicine, Caribou Biosciences, CRISPR therapeutics,...

The prospect of using the popular genome-editing tool CRISPR to treat a host of diseases in people is moving closer to reality.

Medical applications of CRISPRCas9 had a banner year in 2019. The first results trickled in from trials testing the tool in people, and more trials launched. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness.

But although the results of clinical trials of CRISPR genome editing so far have been promising, researchers say that it is still too soon to know whether the technique will be safe or effective in the clinic.

Theres been a lot of appropriate caution in applying this to treating people, says Edward Stadtmauer, an oncologist at the University of Pennsylvania in Philadelphia. But I think were starting to see some of the results of that work.

It has only been seven years since researchers discovered thata molecular defence system called CRISPRCas9, which microbes use to fend off viruses and other invaders, could beharnessed to rewrite human genes.

Since then gene-editing has attracted attention for its potential to modify embryosan application that is ethically and legally fraught if those embryos are destined tobecome human beings. But in parallel, scientists have been testing CRISPR's much less controversial ability to disable or correct problematic genes in other cells in order to treat a host of diseases.

In 2016, Chinese researchers announced that they hadtreated the first person with a CRISPRCas9 therapydesigned to fight cancer. In cells extracted from a participant's blood, the researchers disabled the gene that codes for a protein called PD-1,which holds the immune system in checkbut can shield cancer cells in the process. The scientists then reinjected the cells.

By 2019, the US governments clinicaltrials.gov database listed more than a dozen active studies that are testing CRISPRCas9 as a treatment for a range of diseases from cancer to HIV and blood disorders.

So far, too few people have been treated in these trials to draw any firm conclusions about the safety of CRISPRCas9 therapies or how well they work. Preliminary results from two trialsone in which gene-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancershowed no signs of clinical improvement.

In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. In the man treated for HIV, the researchers attempted to use CRISPR to disable a protein that many strains of HIV use to enter cells. But only 5% of the transplanted cells were editednot enough to cure disease, the researchers reported in September. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University in Beijing and a lead author of the work.

There are early hints that another trial might meet with more success. CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, have treated two people with the genetic disorders sickle-cell anaemia and -thalassaemia. Both deplete oxygen-carrying haemoglobin molecules in the blood: the idea is to use CRISPR to disable a gene that otherwise shuts off production of another form of haemoglobin. Early results suggest that the treatment might have eased some symptoms of the disorders, but the participants will need to be followed for a longer period to be sure.

Other researchers are already itching to move beyond editing cells in a dish. The challenge is in finding ways to transport the gene-editing machinery to where it is needed in the body, says John Leonard, chief executive of Intellia Therapeutics, a biotechnology company in Cambridge, Massachusetts, that is focused on CRISPRCas9 genome editing. The delivery approach is so important.

Last July, the pharmaceutical companies Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin launched a trial to treat the genetic disorder Leber congenital amaurosis 10, which can cause blindness, by editing eye cells. Researchers will inject into the eye a virus containing DNA that encodes the CRISPR genome-editing machinery, bypassing the need to guide those tools through the bloodstream to the specific tissues. The virus will be responsible for carrying the genome-editing tools into cells. It is the first trial to attempt CRISPRCas9 gene editing inside the body, and early results could be reported this year.

That would be a landmark moment for the field, and could pave the way for future trials targeting other organs, says Charles Gersbach, a bioengineer at Duke University in Durham, North Carolina. But he and others say that they hope researchers will eventually move away from using viruses to shuttle genome-editing machinery into cells. Deactivated viruses can still sometimes provoke immune responses, and can only carry a limited amount of DNA.

What's more, some gene-editing tools are currently too large to fit inside commonly used gene-therapy viruses, says chemical biologist Andrew Anzalone at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. These include the souped-up CRISPR systems calledprime editorsthat were first reported in late 2019and might prove to be more precise and controllable than CRISPRCas9.

Intellia is looking for a way around the viruses. The company has partnered with Swiss pharmaceutical giant Novartis to develop fatty nanoparticles that can protect genome-editing molecules as they travel through the bloodstream, but also pass through the membranes of target cells.

These particles tend to accumulate in the liver, and researchers are working to develop particles that infiltrate other tissues, such as muscle or the brain. But for now, Intellia will focus on liver diseases, says Leonard, and plans to launch its first trial of the technology this year. Its crawl before you walk, so to speak, he says.

None of the technologies currently being tested is what researchers envision for the long-term applications of genome-editing, says Gersbach. The approaches that people are taking are the things that we can do today, he says, but not what we would do if we could design the ideal drug.

Leonard says that when he meets with investors, they often demand to know what medical advances will be made in the next six months. We do our best to describe that, but I always end it by saying, Can you imagine a future without gene editing? he says. I have yet to meet the person who says, yes.

This article is reproduced with permission and wasfirst publishedon January 6, 2020.

More here:
Quest to Use CRISPR Against Disease Gains Ground - Scientific American

Bioengineering, once viewed primarily as an academic discipline, is growing up.

Our ability to engineer biology is on the verge of changing the landscape of health and health care. Tools and treatments that are engineered, not discovered CAR-T therapies for cancer, CRISPR for gene editing, stem cell therapies, and more are now making their way not just into new startups but into established industry. Just look at the first-generation CAR-T companies that have been acquired by major biopharma companies, like Bristol-Myers Squibb/Celgene acquiring Juno or Gilead acquiring Kite.

The acquirers, massive organizations built on the foundations of discovery, are now ingesting companies built with engineering DNA. These are two extremely different mindsets. For decades, biopharma companies essentially used scientists to build products, because there was no means to engineer them. The intersection of these worlds is driving us into the future.

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Here come the culture clashes.

These emerge every time major new technologies disrupt an industry. Think of the early days of oil and the evolution as the industry matured from wildcatters one man, one rig, one highly risky and unreliable process to Rockefellers and Rothschilds and a highly sophisticated engineering and discovery process that used every advanced technology and enterprise tool available.

In biopharma and health care today, the old culture of discovery the idea that science is driven by discovering new knowledge (hypothesis > test > repeat) is clashing with the new culture of engineering (design > test > iterate). This clash encompasses how everything is handled, from identifying biological targets to designing clinical trials and even to how we access health care.

Knowing that these clashes are coming will help smooth the way as the biopharma industry integrates bioengineering deeper and more broadly. I see four key clashes worth noting.

We cant yet take for granted a common understanding that we can engineer biology. In spite of bioengineering departments flourishing at revered institutions like Harvard and MIT and Stanford and Berkeley, in spite of the success of new tools like CAR-T and CRISPR, some think that bioengineering is either hype or a passing fad. Thats OK; every new field struggles with the old guard.

Bioengineering still needs to come from a place of clearly and repeatedly explaining its worth with evidence. Lets just get used to this. That said, naysayers are predisposed to cling to old-school approaches. After all, thats the value they have to offer. Ultimately, they will need to adapt to an engineering approach or get engineered out of the process.

The culture of discovery and the culture of engineering value progress differently. Discovery, for example, prioritizes the Eureka! moment above all. One of the most challenging aspects of drug development is that you cant establish a key performance indicator for such moments. Pure discovery is a lottery ticket business that exists in the biopharma industry only because of its incredibly high value and the potential to save millions of lives.

Engineering, on the other hand, values a repeatable process, one that can be stacked or adjusted the way we build with Legos, aiming for compounding results a consistent fractional improvement year over year, leading to exponential improvement over time.

Health care needs both approaches. Todays great discovery will be engineerable tomorrow (OK, maybe 50 years from now). CRISPR, for example, began with discoveries in Haloferax mediterranei, a species of salt-tolerant bacteria. That was pure scientific discovery. But using CRISPR as a tool, as a therapeutic, or as a platform for future innovations is squarely in the world of bioengineering.

The choice of staying or leaving is now at the heart of the debate about what engineering can or cant handle, and what should remain pure, unfettered empirical discovery.

The truth is that engineering can handle empirical approaches. For example, is an A/B test discovery or is it engineering? Its actually both discovery done via an engineering process with iteration. Because biology is so incredibly sophisticated and complex, there will always be discovery risk the risk that some heretofore unknown aspect of biology will lead to failure. But part of engineering is, and should be, handling discovery and failure, and discovery can and should be engineered.

Tools like artificial intelligence and machine learning allow us to introduce to the world of discovery faster throughput, faster iteration, and greater reproducibility. We need to know how and where to apply engineering and discovery frameworks, and where the two worlds meet. Is your discovery risk one where you must wait for serendipity, or can you improve your odds by engineering some part of it?

The discovery and engineering cultures speak something that sounds like the same language, but really isnt. Words like discovery and platform mean very different things in science than they do in engineering. Even success doesnt directly translate: Does it work and we know how we got there, or did we get there in a repeatable process we can tweak?

Getting lucky with a serendipitous discovery is not success in an engineering discipline, nor is getting unlucky a failure in engineering, since you can learn something valuable from failure with which to tweak the process. Like any language issue, we need to recognize the different meanings in those core concepts and know when to use which depending on the world you are in.

Integrating these cultures requires each side to understand core tools, language, and mindsets in both worlds, and knowing where to leverage the differences. Where can discovery yield new empirical information for an engineered process? Where can an engineered process increase the odds of success over a more traditional discovery route?

In health care, as more and more products and tools become engineerable, the world of discovery will need to transition toward integrating engineering. This will be bumpy and uncomfortable and lets be real, there will be blood. But there will also be many bright spots. There will be people trained in science who, when introduced to engineering, feel they can tap into a new world of possibility. There will be engineers who maybe started their careers because as kids they dreamed of engineering trains who suddenly feel they have the potential to help cure cancer.

The audacious dream of engineering biology on a molecular scale is finally being realized not just in practice but commercially if we can surmount the culture clashes.

Vijay Pande, Ph.D., is a general partner at Andreessen Horowitz, a Silicon Valley venture capital firm, and an adjunct professor of bioengineering at Stanford University. He serves on the boards of Apeel Sciences, Asimov, BioAge, Ciitizen, Devoted Health, Freenome, Insitro, Omada, and PatientPing.

Read the original post:
Welcome to the bioengineering culture clash - STAT

What happened

Shares of CRISPR Therapeutics (NASDAQ:CRSP) rose over 113% last year, according to data provided by S&P Global Market Intelligence. The pharma stock built momentum throughout much of the year, but surged in October ahead of an important data presentation that ultimately lived up to the hype. That allowed the gene-editing stock to easily outperform the 28.8% gain of the S&P 500 in 2019.

The end-of-year rally was driven by promising clinical results for its lead drug candidate. The first two individuals, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), dosed with CTX001 achieved functional cures after receiving an initial dose of the gene-editing product. The results need to be proven durable and replicated in a larger number of patients, but the update was about as good as investors could have hoped for at the current stage of development.

Image source: Getty Images.

Both SCD and TDT are caused by structural abnormalities in red blood cells. But these are one of the few cells in the human body that don't contain DNA. That means CRISPR Therapeutics has to harvest stem cells from the bone marrow of patients, apply gene editing to those extracted cells, and then inject the engineered stem cells back into patients (the ex vivo method). If the therapy works, then the engineered stem cells should produce functional red blood cells and potentially result in a cure.

In the early study, the ex vivo approach of CTX001 appeared to do just that. The TDT patient required an average of 16.5 blood transfusions per year in the two years before the clinical trial. Nine months after receiving the gene-editing treatment, the individual was transfusion independent (compared with an expected 12 transfusions) and expressed working copies of hemoglobin on 99.8% of red blood cells.

The SCD patient experienced an average of seven vaso-occlusive crises (painful blockages of blood vessels caused by abnormally shaped red blood cells) per year in the two years before the clinical trial. Four months after receiving the gene-editing treatment, the individual reported no vaso-occlusive crises (compared with an expectation for two such episodes) and expressed working copies of hemoglobin on 94.7% of red blood cells.

The early success of CTX001 bodes well for the ex vivo approach of CRISPR Therapeutics and its partner Vertex Pharmaceuticals(NASDAQ:VRTX), but investors should be careful not to extrapolate the results too broadly. Gene-editing tools that are applied inside the body (in vivo) face significantly steeper obstacles, such as the difficulty of delivering gene-editing payloads to specific tissue types inside the body. There's also the elephant in the room: Scientists are beginning to realize that current-generation CRISPR gene-editing tools don't work all that well.

Nonetheless, CRISPR Therapeutics is the top CRISPR-based gene-editing stock on the market. It has the cash, the partnerships, and the early results to back up its claim to that label.

Read the rest here:
Here's Why CRISPR Therapeutics Stock Jumped 113.2% in 2019 - Motley Fool

The bright wing patterns in butterflies in the genus Heliconius make for a highly visible palette to use CRISPR gene-editing techniques to investigate the evolution of wing patterns and the genes that influence them, say researchers at the Smithsonian Institute of Tropical Research. (Photo credit: Sebastian Menas/Instagram @mena_sebas)

To help shed light on some of these largest questions remaining in evolutionary biology, the authors of a paper published recently in Current Biology paper narrowed in on one evolutionary storyMllerian mimicry in Heliconius butterflies.

Laura Kraft

Heliconius butterflies synthesize compounds from the family of the cyanides and also feed on plants that have these compounds. So, they are very distasteful for predators, especially birds, says Carolina Concha, Ph.D., Biodiversity Genomics Fellow at the Smithsonian Institute of Tropical Research. In order to advertise their toxicity, these butterflies have evolved bright red, orange, yellow and blue warning colors with strong black banding. Unfortunately for the butterflies, birds typically have to bite and kill a few butterflies before learning that bright colors mean bad tastes. So over time, distantly related yet equally distasteful species of these butterflies have converged to have the exact same wing color and pattern so that both convergent species can benefit from an increased warning signal. This causes birds to learn faster by feeding on fewer individuals from either species.

These bright color patterns in Heliconius butterflies have evolved and diverged into a number of species found in Central and South America over the last 12 to 14 million years, but it was only within very recent evolutionary historythe last 2.5-4.5 million yearsthat some distantly related distasteful butterflies converged to have the same patterns. Says Concha, The wing patterns have diversified so much in such a short time, so you have these really diverse forms within a single species and at the same time, you have very distantly related species that converge in a single phenotype.

Carolina Concha, Ph.D., shows off the stack of display boxes of CRISPR-mutation butterflies that she and her collaborators have created in the Smithsonian Institute of Tropical Research in Gamboa, Panama. (Photo credit: Laura Kraft)

CRISPR allows us to make the same butterflies, with one gene missing. Its a way to interrogate nature and just ask What is the function of this gene? and Did it change during evolution?' says Arnaud Martin, author on the Current Biology paper and Assistant Professor at George Washington University. One of the earliest genes to be expressed during butterfly wing development is called WntA (pronounced WIN- tah), and it is thought to be a major gene controlling the paint by number system used by butterflies to color and pattern their wings. WntA was once expressed during embryonic development of many different types of organisms, including vertebrates, but has been lost in most. Perhaps after its role in embryonic development had become downplayed in butterflies, WntA developed a new role in wing patterning, defining the boundaries of black and color banding patterns on adult Heliconius butterfly wings.

The researchers proposed to use CRISPR as scissors to precisely cut out the section of DNA that codes for the WntA gene in a few Mllerian co-mimic pairs. They had two complimentary hypotheses: If mimetic wing patterns developed in different butterflies from using a highly similar gene regulatory mechanism involving WntA, then knocking out the gene should result in the same wing pattern changes in mutant butterflies of the different species. If, on the other hand, identical wing patterns were caused by highly different pathways involving WntA, then the wings of two species of mutant butterflies may exhibit different patterns.

Carolina Concha, Ph.D., carefully injects CRISPR into Heliconius butterfly eggs. If she can inject the eggs within two hours after they are laid, she gets less mosaicism in the CRISPR mutation than with three- or four-hour-old eggs. (Photo credit: Luca Livraghi)

After injecting thousands of eggs, Concha finally got full CRISPR knockout of three co-mimetic pairs of Heliconius butterflies. I honestly thought that co-mimetic Heliconius species were generated by similar tweaks of the same developmental pathways. I was wrong, says Owen McMillan, Ph.D., Dean of Academic Programs at STRI. In all three co-mimetic pairs, the resulting mutant butterflies had dramatically different wing color patterning after removing the WntA gene, supporting the second hypothesis that even highly different pathways can lead to the same wing pattern.

CRISPR allowed us to push our basic understanding of the pathways underlying wing pattern formation in entirely new directions. We have made great progress in identifying the key genes underlying pattern formation, but CRISPR allows us, for the first time, to understand how they work. It is a remarkably cool tool for discovery, says McMillan.

While major changes have been made to the regulatory pathway of WntA, resulting in these wing patterning differences, they do all focus on the same gene. But the really surprising part of this paper is that despite these butterflies developing under different conditions and experiencing different evolutionary histories over millions of years, they still converged upon the same phenotype with a completely different network of gene regulation. This really highlights that the genome has a few favorite genetic tools that drive the evolution of specific parts of the anatomy, but the way these tools can be used is flexible, says Martin

If youd like to learn more, here is a video showing the method of using CRISPR to change butterfly wing patterning:

Laura Kraftis a Ph.D. student at North Carolina State University and a National Science Foundation Graduate Research Fellow. When she isnttraveling the world, she spends her time making science more accessible through science writing and outreach. Email:ljkraft@ncsu.edu.

Related

Read the rest here:
CRISPR Cuts Through Layers of Butterfly Wing-Pattern Evolution - Entomology Today

Dublin, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The "Global CRISPR Technology Market 2019-2025" report has been added to ResearchAndMarkets.com's offering.

The global CRISPR technology market is expected to witness a significant growth rate during the forecast period.

Industry giants are working in CRISPR technology development and are investing in the R&D of the technology. Such investments are expected to create an opportunity for the growth of the market in the near future.

The global CRISPR technology market is segmented on the basis of application and end-user. Based on the application, the market is segmented into biomedical applications, agricultural applications, and industrial applications. In fruit crops, CRISPR technology has numerous applications as it improves the important agronomic traits such as biotic and abiotic stress tolerance and fruit quality.

Further, on the basis of end-user, the market is segmented into pharmaceutical & biopharmaceutical companies, and academic & research institutes. CRISPR being a really new technology seeks the interest of everyone from doctors to academic & research institutes. CRISPR holds a lot of hidden potentials to cure many rare and incurable diseases that are still to be discovered and is driving the academic & research institutes as an end-user segment to grow with a significant rate in the market.

Geographically, the market is segmented into four major regions; North America, Europe, Asia-Pacific and Rest of the world (RoW). Among these, North America is expected to hold a prominent position in the global CRISPR technology market. The presence of major pharma companies in the region tends to enhance the growth of the global CRISPR market.

Further, the report covers the analysis of several players operating in the market. Some of the players include Thermo Fisher Scientific Inc., Merck KGaA, GenScript Biotech Corp., Horizon Discovery Group PLC, CRISPR Therapeutics AG, and others.

The report covers:

Key Topics Covered

1. Report Summary 1.1. Research Methods and Tools1.2. Market Breakdown 1.2.1. By Segments1.2.2. By Geography

2. Market Overview and Insights2.1. Scope of the Report 2.2. Analyst Insight & Current Market Trends2.2.1. Key Findings2.2.2. Recommendations2.2.3. Conclusion2.3. Rules & Regulations

3. Competitive Landscape3.1. Company Share Analysis3.2. Key Strategy Analysis3.3. Key Company Analysis 3.3.1. Thermo Fisher Scientific, Inc. 3.3.1.1. Overview3.3.1.2. Financial Analysis3.3.1.3. SWOT Analysis3.3.1.4. Recent Developments3.3.2. Merck KGaA3.3.2.1. Overview3.3.2.2. Financial Analysis3.3.2.3. SWOT Analysis3.3.2.4. Recent Developments3.3.3. GenScript Biotech Corp.3.3.3.1. Overview3.3.3.2. Financial Analysis3.3.3.3. SWOT Analysis3.3.3.4. Recent Developments3.3.4. Horizon Discovery Group PLC3.3.4.1. Overview3.3.4.2. Financial Analysis3.3.4.3. SWOT Analysis3.3.4.4. Recent Developments3.3.5. CRISPR Therapeutics AG3.3.5.1. Overview3.3.5.2. Financial Analysis3.3.5.3. SWOT Analysis3.3.5.4. Recent Developments

4. Market Determinants4.1. Motivators4.2. Restraints4.3. Opportunities

5. Market Segmentation5.1. CRISPR Technology Market by Application5.1.1. Biomedical Applications5.1.2. Agricultural Applications5.1.3. Industrial Applications5.2. Global CRISPR Technology Market by End-User5.2.1. Pharmaceutical and Biopharmaceutical Companies5.2.2. Academic & Research Institutes

6. Regional Analysis6.1. North America6.1.1. United States6.1.2. Canada6.2. Europe6.2.1. UK6.2.2. Germany6.2.3. Italy6.2.4. Spain6.2.5. France6.2.6. Rest of Europe6.3. Asia-Pacific6.3.1. China6.3.2. India6.3.3. Japan6.3.4. Rest of Asia-Pacific6.4. Rest of the World

7. Company Profiles7.1. AstraZeneca PLC7.2. BASF SE7.3. Beam Therapeutics Inc.7.4. Bio-Rad Laboratories, Inc.7.5. Caribou Bioscience Inc.7.6. Cellectics SA7.7. Cibus, Ltd.7.8. CRISPR Therapeutics AG7.9. Danaher Corp.7.10. Editas Medicine7.11. GeneCopoeia inc.7.12. GenScript Biotech Corp.7.13. Horizon Discovery Group PLC7.14. Intellia Therapeutics Inc.7.15. Lonza Group Ltd.7.16. Merck KGaA7.17. New England Biolabs, Inc.7.18. Origene Technologies, Inc.7.19. Pairwise Plants7.20. Precision Bioscience, Inc.7.21. Sangamo Therapeutics Inc.7.22. Thermo Fisher Scientific, Inc.7.23. Transposagen Biopharmaceuticals, Inc.7.24. Tropic Biosciences UK LTD.7.25. Yield10 Bioscience, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/11g9aw

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CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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CRISPR Technology Market Insights and Forecast, 2019-2025 - Analysis of Biomedical, Agricultural and Industrial Applications - Yahoo Finance

Scientists from China National Rice Research Institute reported that FLN2, a gene that encodes fructokinase-like protein2, influences sugar metabolism as well as rice plants response to salinity stress. The results of their study are published in Biomolecules.

Several mutagenized rice lines were grown under high salinity conditions to pinpoint the genes needed for the expression of salinity tolerance. Some rice lines with mutation in FLN2 showed susceptibility to salinity stress. Wild-type rice lines exposed to salinity stress showed up-regulated FLN2, while CRISPR-Cas9-generated lines with dysfunctional FLN2 exhibited hypersensitivity to salinity stress. Furthermore, sugar metabolism was reduced in the knockout line than in wild-type plants. This may imply that the compromised salinity tolerance in FLN2 knockout plants was caused by the shortage in assimilate needed for growth.

The researchers concluded that FLN2 is vital in seedling growth as well as in tolerance to salinity stress.

Read full, original article: Crop Biotech Update January 8, 2020

More here:
CRISPR-edited crops reveal gene responsible for salt tolerance in rice - Genetic Literacy Project

CRISPR Therapeutics AG (NASDAQ:CRSP) shareholders might be concerned after seeing the share price drop 11% in the last month. But that doesnt undermine the rather lovely longer-term return, if you measure over the last three years. In three years the stock price has launched 170% higher: a great result. To some, the recent share price pullback wouldnt be surprising after such a good run. The fundamental business performance will ultimately dictate whether the top is in, or if this is a stellar buying opportunity.

View our latest analysis for CRISPR Therapeutics

Because CRISPR Therapeutics made a loss in the last twelve months, we think the market is probably more focussed on revenue and revenue growth, at least for now. Generally speaking, companies without profits are expected to grow revenue every year, and at a good clip. Thats because its hard to be confident a company will be sustainable if revenue growth is negligible, and it never makes a profit.

In the last 3 years CRISPR Therapeutics saw its revenue grow at 87% per year. Thats much better than most loss-making companies. Along the way, the share price gained 39% per year, a solid pop by our standards. But it does seem like the market is paying attention to strong revenue growth. Thats not to say we think the share price is too high. In fact, it might be worth keeping an eye on this one.

You can see below how earnings and revenue have changed over time (discover the exact values by clicking on the image).

While the share price may move with revenue, other factors can also play a role. For example, weve discovered 4 warning signs for CRISPR Therapeutics (of which 1 is major) which any shareholder or potential investor should be aware of.

Were pleased to report that CRISPR Therapeutics rewarded shareholders with a total shareholder return of 64% over the last year. That gain actually surpasses the 39% TSR it generated (per year) over three years. The improving returns to shareholders suggests the stock is becoming more popular with time. Shareholders might want to examine this detailed historical graph of past earnings, revenue and cash flow.

If you like to buy stocks alongside management, then you might just love this free list of companies. (Hint: insiders have been buying them).

Please note, the market returns quoted in this article reflect the market weighted average returns of stocks that currently trade on US exchanges.

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

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

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Shareholders Are Thrilled That The CRISPR Therapeutics (NASDAQ:CRSP) Share Price Increased 170% - Simply Wall St

The roaring 20s are upon us, and the investment opportunities set in front of us are exhilarating. This new decade has a lot in store for us with tech as the driving force behind it.

FAANG was the acronym that drove the stock market to continuously new highs over the past decade: Facebook (FB), Amazon (AMZN), Apple (AAPL), Netflix (NFLX), and Google aka Alphabet (GOOGL). This is an acronym that I am sure you are familiar with. These stocks exponential returns may be exhausted, and a new set of equities are ready to take their place. It is time to look for the new FAANG.

When assessing market-shifting companies, you need to look for firms with an exciting product offering characterized by longevity and a substantial total addressable market (TAM). Firms with savvy management teams that are able to navigate through both the best and worst times nimbly.

I have chosen a new acronym of stocks that I believe could change the world in the roaring 20s. The companies include Crispr (CRSP), Sea Limited (SE), Alibaba (BABA), Nvidia (NVDA), and Splunk (SPLK) or SCANS, as I like to call it.

Here I will give a brief introduction of each stock and explain why I believe these shares will drive the market in this new decade.

Sea Limited (SE)

Sea is the leading internet company in Southeast Asia and Taiwan. These economies are digitalizing at an exponential rate, and Sea is well-positioned to take on the quickly expanding addressable market. The company operates three market leader segments, including an ecommerce platform, a digital entertainment division, and a digital payment company (Shopee, Garena, and AirPay, respectively).

The internet economy in Southeast Asia has tripled in the past 5 years to $100 billion and is expected to triple again by 2025 to $300 billion. Sea is growing at an even faster rate, with year-over-year topline appreciation in the high triple-digit percentages as the company continues to take an increasing amount of market share.

Sea Limited is going to be the tech powerhouse that helps turn the third world economies of Southeast Asia and Taiwan into digitalized world markets.

CRISPR (CRSP)

CRISPR is a biomedical firm that is on the verge of changing the world. This company can edit an individuals DNA, an achievement that is going to change modern medicine. This technology could be used to cure almost any disease if it is successfully implemented. What CRISPRs gene therapy does is splice out the bad or disease driving DNA and add healthy strands. The company is also a leader in regenerative stem-cell medicine, which could save the lives of 100s of thousands.

CRISPR has an established portfolio of life-changing therapies in its pipeline at various stages of development. Hemoglobinopathy is the closest to commercially viable and is currently in clinical trials. If it passes clinical trials, I see this stock jumping substantially.

These shares are still a risky asset considering the possibility that none of its gene-therapies make it past the clinical stage. Based on early trials, it appears that the therapy does indeed work, and this potential has begun to be priced into CRSP. The stock has appreciated 350% since it went public in late 2016, and I believe that this is just the beginning of its growth. The ability to change an individuals DNA is going to change the world of medicine.

Alibaba (BABA)

The Amazonof the East has been driving substantial growth, but I dont believe that investors are correctly valuing Alibabas fundamentals. BABA is trading at roughly 1/3rd of Amazons forward P/E valuation (seen below), despite achieving wider margins, stronger profitable, and a greater growth outlook. Alibaba is operating in one of the worlds largest and fastest-growing consumer markets (China).

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Alibaba controls not only Chinas ecommerce market but also its cloud computing space with a 47% market share. Its cloud computing space has the most room to run as Chinas cloud infrastructure continues to expand at an exponential rate high double-digit to triple-digit percentages.

Alibaba still has some geopolitical risk due to the US-China trade war, but as far as this next decades biggest equity drivers, I would replace AZMN with BABA in my portfolio.

Nvidia (NVDA)

This is the most exciting chip maker in the world today. Nvidia is known for the invention of the GPU, which is a chip original purposed for image rendering, but Nvidia has taken its capabilities far beyond this. Nvidias chips are hyper-fast and slowly becoming smarter as the technology develops. Its chips are becoming a necessity in data centers and are an essential element of AI development. I believe that one of Nvidias integrated circuits will be apart of the first true AI, which is going to change the world.

Nvidia is also leveraging 5G with its anticipated cloud gaming platform. Like cloud computing is the future of business data and analytics, cloud gaming is the future of gaming. Nvidia is making a big bet in this field with its cloud platform, GeForce NOW. This platform allows gamers to use their Macs or PCs for gaming anywhere with the high-speed, low-latency technology of Nvidias GPUs without needing Nvidias hardware locally.

Nvidia is undoubtedly a company of the future, and despite its 4-digit gains over the past decade, I believe that this stock still has legs to run. I dont think that the company has scratched the surface of what its chips could do.

Splunk (SPLK)

Splunk is a platform that helps companies utilize real-time machine data for collection, indexing, and alerts, allowing companies to uncover actionable insight from this data no matter the source or format. The company is leveraging AI and machining learning for forecasting and anticipative decision making.

Real-time data management is becoming increasingly necessary in business across industries as this digital age makes speed a competitive advantage. Splunk is well-positioned to take on the massive addressable market that is yet to recruit Splunks services. This firm is well-suited to transform the way our economy utilizes real-time data.

Take Away

The market driving stocks will undoubtedly make excellent long-term investments for the roaring 20s. SCANS will be a force to be reckoned with in this next decade. Short term volatility in these stocks shouldnt cause you to shy away from their long-term potential. I believe we may be on the edge of a market correction, so if you are worried about short-term earnings, I may wait for a pullback. If you are a long-term investor that is willing to ride this decades waves, I wouldnt hesitate to pull the trigger on these stocks.

Breakout Biotech Stocks with Triple-Digit Profit Potential

The biotech sector is projected to surge beyond $775 billion by 2024 as scientists develop treatments for thousands of diseases. Theyre also finding ways to edit the human genome to literally erase our vulnerability to these diseases.

Zacks has just released Century of Biology: 7 Biotech Stocks to Buy Right Now to help investors profit from 7 stocks poised for outperformance. Our recent biotech recommendations have produced gains of +98%, +119% and +164% in as little as 1 month. The stocks in this report could perform even better.

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Click to get this free report Splunk Inc. (SPLK) : Free Stock Analysis Report Sea Limited Sponsored ADR (SE) : Free Stock Analysis Report NVIDIA Corporation (NVDA) : Free Stock Analysis Report Netflix, Inc. (NFLX) : Free Stock Analysis Report Alphabet Inc. (GOOGL) : Free Stock Analysis Report Facebook, Inc. (FB) : Free Stock Analysis Report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report Alibaba Group Holding Limited (BABA) : Free Stock Analysis Report Amazon.com, Inc. (AMZN) : Free Stock Analysis Report Apple Inc. (AAPL) : Free Stock Analysis Report To read this article on Zacks.com click here. Zacks Investment Research

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SNACS: The FAANG Of The Roaring 20s - Yahoo Finance