Archive for the ‘Crispr’ Category

Watching some recent stock price activity for CRISPR Therapeutics AG (:CRSP), we have seen shares trading near the $47.7 level. Investors have a wide range of tools at their disposal when undertaking stock research. Investors will often monitor the current stock price in relation to its 52-week high and low levels. The 52-week high is currently $52.56, and the 52-week low is presently $22.73. When the current stock price is trading close to either the 52-week high or 52-week low, investors may pay increased attention to see if there will be a breakthrough that level. Taking a look at some previous stock price activity, we can see that shares have moved 66.96% since the beginning of the year. Pulling the focus closer to the last 4 weeks, shares have seen a change of 2.12%. Over the past 5 trading days, the stock has moved -3.97%.Over the past 12 weeks, the stock has seen a change of -0.02%.

Investors may be searching high and low for the next breakout winner in the stock market. As companies continue to release quarterly earnings reports, investors will be looking for stocks that have the potential to move to the upside in the coming months. Tracking earnings can be a good way for investors to see how the company is stacking up to analyst estimates. Some investors prefer to track sell-side estimates very closely. Others prefer to do their own research and make their own best guesses on what the actual numbers will be. A solid earnings beat may help ease investor worries if the stock has been underperforming recently. On the flip side, a bad earnings miss may cause investors to take a much closer look at what the future prospects look like for the company.

Investors might be paying attention to what Wall Street analysts think about shares of CRISPR Therapeutics AG (:CRSP). Taking a peek at the current consensus broker rating, we can see that the ABR is 2.07. This average rating is provided by Zacks Research. This simplified numeric scale spans the range of one to five which translates brokerage firm Buy/Sell/Hold recommendations into an average broker rating. A low number in the 1-2 range typically indicates a Buy, 3 indicates a Hold and 4-5 represents a consensus Sell rating. In terms of the number of analysts that have the stock rated as a Buy or Strong Buy, we can see that the number is currently 9.

Shifting the focus to some earnings data, we have noted that the current quarter EPS consensus estimate for CRISPR Therapeutics AG (:CRSP) is -0.95. This EPS estimate consists of 6 Wall Street analysts taken into consideration by Zacks Research. For the previous reporting period, the company posted a quarterly EPS of -1.01. Sell-side analysts often provide their best researched estimates at what the company will report. These estimates hold a lot of weight on Wall Street and the investing community. Sometimes these analyst projections are spot on, and other times they are off. When a company reports actual earnings results, the surprise factor can cause a stock price to fluctuate. Investors will often pay added attention to a company that has beaten estimates by a large margin.

Looking at some analyst views on shares of CRISPR Therapeutics AG (:CRSP), we note that the consensus target price is resting at $67.94. This is the consensus target using estimates provided by the covering analysts polled. Sell-side analysts often produce target estimates for the companies that they track closely. Price target estimates can be calculated using various methods, and this may cause some analyst estimates to be drastically different than others. Many investors will track stock target prices, especially when analysts update the target price projections.

Investors have various approaches they can take when deciding what stocks to stuff the portfolio with. Some investors may choose to use fundamental analysis, and some may choose to use technical analysis. Others may employ a combination of the two approaches to make sure no stone is left unturned. Investors looking for bargains in the market may be on the lookout for the stock that offers the best value. This may involve finding stocks that have fallen out of favor with the overall investing community but still have low PE ratios and higher dividend yields. Whatever approach is used, investors may benefit greatly from making sure that all the homework is done, and all of the angles have been examined properly.

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Analyst Views in Focus on Shares of CRISPR Therapeutics AG (:CRSP) - Blackwell Bulletin

BERKELEY, Calif., Sept. 24, 2019 /PRNewswire/ -- Today, the U.S. Patent and Trademark Office (USPTO) granted a new CRISPR-Cas9 patent to the University of California (UC), University of Vienna, and Dr. Emmanuelle Charpentier, bringing new compositions and methods to the continuously expanding patent portfolio. U.S. Patent 10,421,980 covers compositions of certain DNA-targeting RNAs that contain RNA duplexes of defined lengths that hybridize with Cas9 and target a desired DNA sequence.

The patent also covers methods of targeting and binding a target DNA, modifying a target DNA, or modulating transcription from a target DNA wherein the method comprises contacting a target DNA with a complex that includes a Cas9 protein and a DNA-targeting RNA.

In September, several patents have been issued to UC, increasing its U.S. CRISPR-Cas9 portfolio to 15 patents. In the coming months, based on applications allowed by the USPTO, UC's portfolio will total 18 patents, covering compositions and methods for the CRISPR-Cas9 gene-editing technology, including targeting and editing genes and modulating transcription in any setting, such as within plant, animal, and human cells.

"With every patent that issues, UC strengthens its position as the leader in CRISPR-Cas9 intellectual property in the United States," said Eldora L. Ellison, Ph.D., lead patent strategist on CRISPR-Cas9 matters for UC and a Director at Sterne, Kessler, Goldstein & Fox. "We are steadfast in our commitment to developing a comprehensive patent portfolio that protects the groundbreaking work of the Doudna-Charpentier team on CRISPR-Cas9."

The Doudna-Charpentier team that invented the CRISPR-Cas9 DNA-targeting technology included Jennifer Doudna and Martin Jinek at the University of California, Berkeley; Emmanuelle Charpentier (then of Umea University); and Krzysztof Chylinski at the University of Vienna. The compositions and methods covered by today's patent, as well as the other compositions and methods claimed in UC's previously issued patents and those set to issue, were included among the CRISPR-Cas9 gene editing technology work disclosed first by the Doudna-Charpentier team in its May 25, 2012 priority patent application.

Additional CRISPR-Cas9 patents in this team's portfolio include 10,000,772; 10,113,167; 10,227,611; 10,266,850; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,358,658; 10,358,659; 10,385,360; 10,400,253; 10,407,697; and 10,415,061. These patents are not a part of the PTAB's recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broad's CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 15 patents granted in the U.S. so far. The European Patent Office (representing more than 30 countries), as well as patent offices in the United Kingdom, China, Japan, Australia, New Zealand, Mexico, and other countries, have issued patents for the use of CRISPR-Cas9 gene editing in all types of cells.

University of California has a long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind. Consistent with its open-licensing policies, UC allows nonprofit institutions, including academic institutions, to use the technology for non-commercial educational and research purposes.

In the case of CRISPR-Cas9, UC has also encouraged widespread commercialization of the technology through its exclusive license with Caribou Biosciences, Inc. of Berkeley, California. Caribou has sublicensed this patent family to numerous companies worldwide, including Intellia Therapeutics, Inc. for certain human therapeutic applications. Additionally, Dr. Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Limited.

View original content:http://www.prnewswire.com/news-releases/uspto-awards-15th-us-crispr-cas9-patent-to-university-of-california-300923678.html

SOURCE University of California Office of the President

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USPTO awards 15th U.S. CRISPR-Cas9 patent to University of California - P&T Community

Integration of CRISPR-case9 technology to accelerate the discovery of innovative antibiotics

DEINOVE (Euronext Growth Paris: ALDEI), a French biotechnology company that relies on a radical innovation approach to develop innovative antibiotics and bio-sourced active ingredients for cosmetics and nutrition, announces that it has expanded its technological platform with an advanced genetic tool, the CRISPR-cas9 system, to enhance its ability to optimize various microorganisms.

In the last few years, DEINOVE has set up a high throughput genetic engineering platform specifically dedicated to rare microorganisms and thus demonstrated its ability to adapt genetic tools to poorly described organisms. Thus, the exploitation of Deinococci as microbial plants has allowed the large-scale production of pure high value-added compounds such as carotenoids. It should be recalled that Deinococci are extremophilic microorganisms whose biological and molecular specificities have so far been little studied and therefore unexploited.

After developing a platform dedicated to the identification of novel antibiotic structures produced by rare bacteria (AGIR Program), DEINOVE strengthens its expertise in genetic engineering with the integration of a cutting-edge tool, the CRISPR-cas9 technology, known as molecular scissors, which has revolutionized genetic engineering in recent years.

The objective for DEINOVE is to be able to directly manipulate the strains producing antimicrobial activities or to transfer these activities into phylogenetically close frames. This has been successfully achieved by the Company which has made the Streptomyces chassis an effective producer of a pharmaceutical intermediate initially produced by Microbacterium arobescens (proof of concept DNB101/102).

Genome editing occurs at two levels. First, highlights the cluster of genes at the origin of the antibiotic activity of interest. To optimize the spectrum of activity and eliminate any potential cytotoxicity, the structure of a natural molecule can then be modified by directly, finely and precisely editing the genes responsible for this activity.

This technology opens up many opportunities in the identification and optimized production of new antibiotic structures.

"Our expertise in the genetic engineering of a variety of microorganisms, unusual for some, is unique, and the integration of CRISPR-cas9 extends the possibilities of our platform," says Georges GAUDRIAULT, Scientific Director of DEINOVE. "We continue to structure the various technological bricks of the AGIR platform to be able to drastically accelerate the identification and optimization of new antibiotic structures. This technology is an additional asset in our race against the clock in the face of rising antimicrobial resistance."

ABOUT DEINOVE

DEINOVE is a French biotechnology company, a leader in disruptive innovation, which aims to help meet the challenges of antibiotic resistance and the transition to a sustainable production model for the cosmetics and nutrition industries.

DEINOVE has developed a unique and comprehensive expertise in the field of rare bacteria that it can decipher, culture, and optimize to disclose unsuspected possibilities and induce them to produce biobased molecules with activities of interest on an industrial scale. To do so, DEINOVE has been building and documenting since its creation an unparalleled biodiversity bank that it exploits thanks to a unique technological platform in Europe.

DEINOVE is organized around two areas of expertise:

Within the Euromedecine science park located in Montpellier, DEINOVE employs 60 employees, mainly researchers, engineers, and technicians, and has filed more than 350 patent applications internationally. The Company has been listed on EURONEXT GROWTH since April 2010.

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Integration of CRISPR-case9 technology to accelerate the discovery of innovative antibiotics - Yahoo Finance

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

DEINOVE (Paris:ALDEI) (Euronext Growth Paris: ALDEI), a French biotechnology company that relies on a radical innovation approach to develop innovative antibiotics and bio-sourced active ingredients for cosmetics and nutrition, announces that it has expanded its technological platform with an advanced genetic tool, the CRISPR-cas9 system, to enhance its ability to optimize various microorganisms.

In the last few years, DEINOVE has set up a high throughput genetic engineering platform specifically dedicated to rare microorganisms and thus demonstrated its ability to adapt genetic tools to poorly described organisms. Thus, the exploitation of Deinococci as microbial plants has allowed the large-scale production of pure high value-added compounds such as carotenoids. It should be recalled that Deinococci are extremophilic microorganisms whose biological and molecular specificities have so far been little studied and therefore unexploited.

After developing a platform dedicated to the identification of novel antibiotic structures produced by rare bacteria (AGIR Program), DEINOVE strengthens its expertise in genetic engineering with the integration of a cutting-edge tool, the CRISPR-cas9 technology, known as molecular scissors, which has revolutionized genetic engineering in recent years.

The objective for DEINOVE is to be able to directly manipulate the strains producing antimicrobial activities or to transfer these activities into phylogenetically close frames. This has been successfully achieved by the Company which has made the Streptomyces chassis an effective producer of a pharmaceutical intermediate initially produced by Microbacterium arobescens (proof of concept DNB101/102).

Genome editing occurs at two levels. First, highlights the cluster of genes at the origin of the antibiotic activity of interest. To optimize the spectrum of activity and eliminate any potential cytotoxicity, the structure of a natural molecule can then be modified by directly, finely and precisely editing the genes responsible for this activity.

This technology opens up many opportunities in the identification and optimized production of new antibiotic structures.

"Our expertise in the genetic engineering of a variety of microorganisms, unusual for some, is unique, and the integration of CRISPR-cas9 extends the possibilities of our platform," says Georges GAUDRIAULT, Scientific Director of DEINOVE. "We continue to structure the various technological bricks of the AGIR platform to be able to drastically accelerate the identification and optimization of new antibiotic structures. This technology is an additional asset in our race against the clock in the face of rising antimicrobial resistance."

ABOUT DEINOVE

DEINOVE is a French biotechnology company, a leader in disruptive innovation, which aims to help meet the challenges of antibiotic resistance and the transition to a sustainable production model for the cosmetics and nutrition industries.

DEINOVE has developed a unique and comprehensive expertise in the field of rare bacteria that it can decipher, culture, and optimize to disclose unsuspected possibilities and induce them to produce biobased molecules with activities of interest on an industrial scale. To do so, DEINOVE has been building and documenting since its creation an unparalleled biodiversity bank that it exploits thanks to a unique technological platform in Europe.

DEINOVE is organized around two areas of expertise:

Within the Euromedecine science park located in Montpellier, DEINOVE employs 60 employees, mainly researchers, engineers, and technicians, and has filed more than 350 patent applications internationally. The Company has been listed on EURONEXT GROWTH since April 2010.

Visit http://www.deinove.com

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DEINOVE: Integration of CRISPR-case9 Technology to Accelerate the Discovery of Innovative Antibiotics - Business Wire

A study has shown that CRISPR can be used as a regenerative technique to treat Duchenne muscular dystrophy, which could be developed as a therapeutic option for humans.

Researchers have successfully demonstrated in a mouse model that CRISPR can regenerate muscle suffering from Duchenne muscular dystrophy (DMD). They believe that with more study, their method may be used to treat children with the condition.

The study was led by the University of Missouri School of Medicine, US in collaboration with other researchers. Previous research has shown that children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin.

If we can correct the mutation in muscle stem cells, then cells regenerated from edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells, said Dr Dongsheng Duan, Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and the senior author of the study.

The researchers first delivered the gene editing tools to immune-deficient mouse muscle through a viral vector known as AAV9. They observed that the transplanted muscle died first, then regenerated from its stem cells, which carried the edited gene.

Previous research has shown that children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin

Next, they tested their method in a mouse model of DMD. The stem cells in the diseased muscle were edited and produced dystrophin.

This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases, Duan said. Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout a patients life.

The results were published in Molecular Therapy.

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CRISPR used to treat Duchenne muscular dystrophy in mice - Drug Target Review

The hubris of some scientists knows no bounds. Less than a year after He Jiankui, a Chinese biophysicist, drew scorn and censure for creating gene-edited twins, Denis Rebrikov, a Russian molecular biologist, boldly announced his plan to follow in Hes genome editing footsteps. Rebrikovs initial stated goal for his proposed research was to prevent the transmission of HIV from infected women to their offspring, though he later suggested other targets, including dwarfism, deafness, and blindness.

In 1998, Nobel laureate Mario Capecchi suggested that resistance to HIV infection was a genetic enhancement that might appeal to potential parents. Twenty years later, in November 2018, He revealed his use of CRISPR-Cas9 genome editing technology to disable a gene called CCR5 in an attempt to create children with resistance to HIV.

Hes research activities were known to a number of senior American scientists, all of whom elected to remain silent about his work. It was only after the twins birth that the world learned of this secret science. Matthew Porteus, one of the scientists who was complicit in the silence, summarized his promise of confidentiality to He this way:

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Youre a scientist talking to a scientist. Our culture is that you respect confidentiality and that when people reveal things in confidence to you, you respect that confidence. And I said, well, Im not going to publicly discuss what you just told me because that is for you to publicly discuss.

A groundswell of condemnation followed Hes public announcement of the twins birth. There was pointed criticism from Feng Zhang, one of the co-discoverers of the CRISPR-Cas9 genome editing technology, and from David Baltimore, who co-chaired international summits of human genome editing in 2015 and 2018. Quoting from the first International Summit Statement, Zhang and Baltimore independently affirmed that the experiment was irresponsible given the lack of data confirming the safety and effectiveness of using CRISPR in humans, as well as the absence of broad societal consensus.

Members of the organizing committee for the 2018 International Summit on Human Genome Editing where He first presented some of the details of his research described the experiment as irresponsible and said it failed to meet international norms. The committee did not, however, reaffirm the position outlined in the 2015 Summit Statement that [i]t would be irresponsible to proceed with any clinical use of germline editing unless and until: (i) the relevant safety and efficacy issues have been resolved, based on an appropriate understanding and balancing of risks, potential benefits, and alternatives, and (ii) there is broad societal consensus about the appropriateness of the proposed application. Instead, the committee concluded that heritable genome editing could be acceptable in the future and suggested that it was time to define a rigorous responsible translational pathway toward such trials.

This shift in orientation is particularly noteworthy when considering the following. In 2015, a researcher performed genome editing on non-viable human embryos that did not involve the transfer of edited embryos to a woman for reproduction. The first summit organizing committee determined that heritable genome editing research was irresponsible unless and until In 2018, He performed genome editing on viable human embryos and transferred these edited embryos to a woman who gave birth to gene-edited children, yet the second summit organizing committee asserted the need for a responsible pathway forward.

Several authors of the 2015 Summit Statement, myself included, disagreed with the position taken by the authors of the 2018 Summit Statement. Along with others, including two of the three CRISPR pioneers Emmanuelle Charpentier and Feng Zhang we issued a call in March 2019 to adopt a moratorium on heritable genome editing. Jennifer Doudna, the other CRISPR pioneer, expressly declined to participate in this initiative.

We reiterated the importance of dialogue within and across nations, and the need for broad societal consensus on the appropriateness of altering the human genome for a particular purpose before any such research could proceed. The purpose of the proposed global moratorium was to provide time for careful study of the relevant technical and ethical issues to determine whether to pursue heritable human genome editing and, if that question were answered in the affirmative, to then determine how to proceed with making modifications to the human genome.

The whether of heritable human genome editing has not been resolved, and yet some scientists continue to race ahead with the how of it, essentially ignoring the myriad calls for public consultation. To be sure, other scientists are willing to heed the call, but would prefer to limit public consultation to public education.

I dont agree with this position. As I write in a new book, Altered Inheritance, we need to move the dial from public education (which typically is limited to talking at the public), to public engagement (which necessarily involves listening to the public), and then on to public empowerment (which is about shared decision-making).

To this end, we need slow science. Science needs time to think and to digest. Time is also needed to promote ethics literacy and to facilitate broad societal consensus where the goal is unity, not unanimity. Decision-making by consensus is about engaged, respectful dialogue and deliberation, where all participants recognize at the outset that knowledge is value laden; that we can and should learn from each other; and that no one should impose his or her will on others.

Metaphorically speaking, the human genome belongs to all of us. So we should all have a say in whether to proceed with making heritable changes to our shared genome. Decision-making by consensus, which begins with outreach and openness, is a means to this end. The goal is to create an environment in which all positions (not all persons) can be heard and understood, and in which there are reasonable opportunities for integrity-preserving compromises in pursuit of the common good. The underlying values are inclusivity, responsibility, self-discipline, respect, co-operation, struggle, and benevolence.

Scientists can meaningfully contribute to consensus building around genome editing. As individuals and as committee members, for example, they can effectively serve the common good by helping policymakers, legislators, and members of the public better align scientific information and opportunities with discrete values and interests.

I wrote Altered Inheritance as a call to action. It is a call for scientists to slow down, to reflect deeply on their science and their priorities, and to find meaningful ways to contribute to science policy in pursuit of the common good. It is also a call for all of us to take collective responsibility for the biological and social future of humankind as we think carefully about what kind of world we want to live in, and how genome editing technology might help us build that world.

Franoise Baylis is University Research Professor at Dalhousie University in Halifax, Nova Scotia, and author of Altered Inheritance: CRISPR and the Ethics of Human Genome Editing (Harvard University Press, September 2019).

Link:
Genome editing needs a dose of slow science - STAT

As a growing population and climate change threaten food security, researchers around the world are working to overcome the challenges that threaten the dietary needs of humans and livestock. A pair of scientists is now making the case that the knowledge and tools exist to facilitate the next agricultural revolution we so desperately need.

Cold Spring Harbor Laboratory (CSHL) Professor Zach Lippman, a Howard Hughes Medical Institute investigator, recently teamed up with Yuval Eshed, an expert in plant development at the Weizmann Institute of Science in Israel, to sum up the current and future states of plant science and agriculture.

Their review, published in Science, cities examples from the last 50 years of biological research and highlights the major genetic mutations and modifications that have fueled past agricultural revolutions. Those include tuning a plants flowering signals to adjust yield, creating plants that can tolerate more fertilizer or different climates, and introducing hybrid seeds to enhance growth and resist disease.

Beneficial changes like these were first discovered by chance, but modern genomics has revealed that most of them are rooted in two core hormonal systems: Florigen, which controls flowering; and Gibberrellin, which influences stem height.

Lippman and Eshed suggest that in an age of fast and accurate gene editing, the next revolutions do not need to wait for chance discoveries. Instead, by introducing a wide variety of crops to changes in these core systems, the stage can be set to overcome any number of modern-day challenges.

Dwarfing and flower power revolutions

To explain their point, the scientists reviewed research that focused on key moments in agricultural history, such as the Green Revolution.

Before the 1960s, fertilizing for a large wheat yield would result in the plants growing too tall. Weighed down with their grainy bounty, the wheat stems would fold and rot away, resulting in yield losses. It was only after Nobel laureate Norman Borlaug began working with mutations that affect the Gibberellin system that wheat became the shorter and reliable crop we know today. Borlaugs dwarfing was also applied to rice, helping many fields weather storms that would have been catastrophic only years before. This reapplication of the same technique to a different plant hinted that a core system was in play.

More recent examples Lippman and Eshed mention include the changes undergone by cotton crops in China. There, growers turned the normally sprawling, southern plantation plant into a more compact, faster flowering bush better suited for Chinas northern climate. To do so, they took advantage of a mutation that affects Florigen, which promotes flowering, and its opposite, Antiflorigen.

This kind of change is related to Lippmans works. He often works with tomatoes and explained that an Antiflorigen mutation in tomato was also the catalyst that transformed the Mediterranean vine crop into the stout bushes grown in large-scale agricultural systems throughout the world today. Whats striking, Lippman said, is that cotton is quite unlike any tomato.

Theyre evolutionary very different in terms of the phylogeny of plants. And despite that, what makes a plant go from making leaves to making flowers is the same, he said. That core program is deeply conserved.

Fine-tuning a revolution

As the review details, this has defined what makes an agricultural revolution. A core system either Gibberellin, Florigen, or both is affected by a mutation, resulting in some helpful trait. In a moment of pure serendipity, the plants boasting this trait are then discovered by the right person.

It then takes many more years of painstaking breeding to tweak the intensity of that mutation until it affects the system just right for sustainable agriculture. Its like tuning an instrument to produce the perfect sound.

Lippman and Eshed note that CRISPR gene editing is speeding up that tuning process. However, they show that the best application of gene editing may not be to just tune preexisting revolutionary mutations, but instead, to identify or introduce new ones.

If past tuning has been creating genetic variation around those two core systems, maybe we can make more variety within those systems, he said. It would certainly mitigate the amount of effort required for doing that tuning, and has the potential for some surprises that could further boost crop productivity, or adapt crops faster to new conditions.

A future in chickpeas?

More of that genetic variety could also set the stage for new agricultural revolutions. By introducing genetic variation to those two core systems that define most revolutions, farmers might get to skip the serendipitous waiting game. Chickpea is one example.

Theres a lot more room for us to be able to create more genetic diversity that might increase productivity and improve adaptation survival in marginal grounds, like in drought conditions, Lippman said.

Drought resistance is just one benefit of under-utilized crops. Past revolutions have allowed crops to be more fruitful or to grow in entirely new hemispheres. Having a means to continue these revolutions with more crops and at a greater frequency would be a boon in a crowded, hungry, and urbanizing world.

Given that rare mutations of Florigen/Antiflorigen and Gibberellin/DELLA mutations spawned multiple revolutions in the past, it is highly likely that creating novel diversity in these two hormone systems will further unleash agricultural benefits, the scientists wrote.

Original article: The next agricultural revolution is here

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CRISPR gene editing poised to streamline next 'agricultural revolution,' plant scientists say - Genetic Literacy Project

Organic grower groups on Sept. 17 wrote they are strongly opposed to opening a formal dialogue about allowing gene-editing in organic agriculture.

A letter from the Organic Farmers Association (OFA), was signed by 79 organic farm organizations and sent to Secretary Sonny Perdue and other top officials and lawmakers.

Introducing any dialogue about any form of genetic engineering into organics would be a major distraction for the USDA NOP and the National Organic Standards Board, Kate Mendenhall, director of OFA, said in a press release. We have crucial issues in organic agriculture that need the Departments full attention, such as stopping organic import fraud, closing certification loopholes, enforcing our current organic standards equitably and uniformly, and updating obsolete database technology.

Gene editing and all other forms of genetic engineering are currently prohibited under the guidelines of organic certification. The letter came in response to an earlier statement by Department Undersecretary Greg Ibach concerning opening a dialogue about gene-editing in organic agriculture.

Read full, original article: Organic growers: Gene-editing dialogue a bad idea

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Organic Farmers Association rejects USDA offer to discuss benefits of CRISPR gene editing - Genetic Literacy Project

Geneticengineering. I realize that this topic has been beaten to death in popularculture, but I dont think the focus has been on the actual technologyreallyonly the flashy outcomes for lay people. I can understand the need to simplifyand sensationalize for entertainment, but decoupling the effects from the causeis, at best, ignorant and, at worst, misleading.

The reason that genetic engineering is populartoday is largely because of the discovery of CRISPR. But its important to notethat the field itself is not new; nearly all commercial forms of insulin arefrom genetically engineered bacteria.

Prior to Clustered Regularly InterspacedShort Palindromic Repeats (CRISPR), technologies like Zinc Finger Nucleases(ZFNs) were somewhat random. While it was likely that the gene you wanted tomanipulate would be inserted into a specific location, it was unclear where inthe hosts DNA it would end up. Far more often than not, the gene would end upeither in the middle of another host gene (likely lethal) or end up in thejunkyard of the host genome, which is effectively useless. Both problemseffectively made genetic engineering on humans far too risky.

The introduction of CRISPR, however, hascompletely changed the field.

CRISPR works similarly to ZFNs, exceptthat it has a very specific targeting domain so that the genes almost alwaysend up in the location that you want them to. While there are still minor kinksto correct, the technique will likely be perfected within this decade. Whilethis technique is no doubt one of the finest inventions in the field ofbiology, even the person that discovered it, Dr. Jennifer Doudna, is callingfor the halting of research in the field until bioethics has a chance to catchup.

The terms designer babies and genedrive are very common buzzwords; however, they genuinely do present ethicalchallenges for us a species. For example, most people wouldnt have a problemusing CRISPR to eradicate debilitating genetic conditions or destroying theability of insect-carried diseases to infect people.

The problem arises when we begin toconsider what counts as pathology, there is an argument that variation fromsocietal, social or biological normality makes people unique. Surely somethinglike schizophrenia or leukemia is morally permissible to eradicate, but whatabout autism, homosexuality or intersexuality?Its a relatively short slippery slope before you end up at eugenics.

Another cause for concern is theecological impact of transgenics. Using the CRISPR based Gene Drive construct,you can force all offspring of a transgenic organism to carry your gene andtheir offspring, and then their offspring. This is ideal in a lab; however, ifa single individual is accidentally released into the environment, it could easilydamage genetic diversity, and permanently disturb the careful equilibrium of anecosystem.

There are instances in which not usingcheap, readily available technology like CRISPR to cure or prevent diseases maybe unethical. For example, the technology to destroy the means by which malariaspreads already exists. Is it really ethical to allow a disease that affectsover 200 million people a year (90% of whom are children) to exist? Are therelimits that we shouldnt cross? Until we have those discussions and draw thelines, research in genetic engineering is effectively playing with fire,analogous to research in nuclear fission during the Cold War.

Like a thermonuclear bomb, releasingCRISPR technology into the world, whether using it for humans or other animals,is not an action that we can reverse, and its results could be equallycatastrophic to life on earth.

These discussions arent entirelyhypothetical by the way; the first genetically modified human babies were bornin China last year.

To clarify, I am not against progress inCRISPR research. I am a huge fan of the technology and I believe it can be aninvaluable resource to improve the world. However, as a student in this field,I am concerned with the ramifications of this techology, enough that it givesme pause. The public discussion surrounding genetic engineering and legislationdesperately needs to catch up to the science.

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Genetic engineering and the end of the world - The Medium

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Companies which are transforming market are: Thermofisher Scientific, CRISPR Therapeutics, Editas Medicine, NHGRI, Intellia Therapeutics, Merck KGaA, Horizon,

Product segment analysis of the market covers: Zinc finger nucleases (ZFNs), Transcription Activator-Like Effector-based Nucleases (TALENs), CRISPR-Cas system

Applications of the market are: Sickle Cell Disease, Heart Disease, Diabetes, Alzheimers Disease, Obesity, Others

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In the next part of the global Gene Editing Tools market report, analytical analysis of the subject matter and adequate survey data makes the reports explicitly beneficial. Analysis of upstream raw materials, downstream demand and current market dynamics is also carried out. The conclusion part of this report offers the new project, key development areas, business overview, product/services specification, SWOT analysis, investment feasibility analysis, return analysis, and development trends.In addition, supply, and consumption are studied along with the gap between them. Import and export statistics are also given in this part and finally trade and distribution analysis has been provided.

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Global Gene Editing Tools Market 2019 Growth Analysis Thermofisher Scientific, CRISPR Therapeutics, Editas Medicine, NHGRI, Intellia Therapeutics -...

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Caribou Biosciences, Integrated DNA Technologies (IDT), CRISPR Therapeutics, Merck, Mirus Bio, Editas Medicine, Takara Bio, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, Agilent Technologies, Cellecta, GenScript, GeneCopoeia, Synthego

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Biotechnology Companies, Pharmaceutical Companies, Academic Institutes, Research and Development Institutes

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CRISPR Cas9 Market Outlook -Industry Growth Factors, Market Revenue and More - Stock Market Pioneer

The hubris of some scientists knows no bounds. Less than a year after He Jiankui, a Chinese biophysicist, drew scorn and censure for creating gene-edited twins, Denis Rebrikov, a Russian molecular biologist, boldly announced his plan to follow in Hes genome editing footsteps. Rebrikovs initial stated goal for his proposed research was to prevent the transmission of HIV from infected women to their offspring, though he later suggested other targets, including dwarfism, deafness, and blindness.

In 1998, Nobel laureate Mario Capecchi suggested that resistance to HIV infection was a genetic enhancement that might appeal to potential parents. Twenty years later, in November 2018, He revealed his use of CRISPR-Cas9 genome editing technology to disable a gene called CCR5 in an attempt to create children with resistance to HIV.

Hes research activities were known to a number of senior American scientists, all of whom elected to remain silent about his work. It was only after the twins birth that the world learned of this secret science. Matthew Porteus, one of the scientists who was complicit in the silence, summarized his promise of confidentiality to He this way:

Youre a scientist talking to a scientist. Our culture is that you respect confidentiality and that when people reveal things in confidence to you, you respect that confidence. And I said, well, Im not going to publicly discuss what you just told me because that is for you to publicly discuss.

Read more: He Jiankui tried to protect CRISPR babies against HIV. But his attempted fix shortens lives, study shows

A groundswell of condemnation followed Hes public announcement of the twins birth. There was pointed criticism from Feng Zhang, one of the co-discoverers of the CRISPR-Cas9 genome editing technology, and from David Baltimore, who co-chaired international summits of human genome editing in 2015 and 2018. Quoting from the first International Summit Statement, Zhang and Baltimore independently affirmed that the experiment was irresponsible given the lack of data confirming the safety and effectiveness of using CRISPR in humans, as well as the absence of broad societal consensus.

Members of the organizing committee for the 2018 International Summit on Human Genome Editing where He first presented some of the details of his research described the experiment as irresponsible and said it failed to meet international norms. The committee did not, however, reaffirm the position outlined in the 2015 Summit Statement that [i]t would be irresponsible to proceed with any clinical use of germline editing unless and until: (i) the relevant safety and efficacy issues have been resolved, based on an appropriate understanding and balancing of risks, potential benefits, and alternatives, and (ii) there is broad societal consensus about the appropriateness of the proposed application. Instead, the committee concluded that heritable genome editing could be acceptable in the future and suggested that it was time to define a rigorous responsible translational pathway toward such trials.

Story continues

This shift in orientation is particularly noteworthy when considering the following. In 2015, a researcher performed genome editing on non-viable human embryos that did not involve the transfer of edited embryos to a woman for reproduction. The first summit organizing committee determined that heritable genome editing research was irresponsible unless and until In 2018, He performed genome editing on viable human embryos and transferred these edited embryos to a woman who gave birth to gene-edited children, yet the second summit organizing committee asserted the need for a responsible pathway forward.

Several authors of the 2015 Summit Statement, myself included, disagreed with the position taken by the authors of the 2018 Summit Statement. Along with others, including two of the three CRISPR pioneers Emmanuelle Charpentier and Feng Zhang we issued a call in March 2019 to adopt a moratorium on heritable genome editing. Jennifer Doudna, the other CRISPR pioneer, expressly declined to participate in this initiative.

Read more: The CRISPR shocker: How genome-editing scientist He Jiankui rose from obscurity to stun the world

We reiterated the importance of dialogue within and across nations, and the need for broad societal consensus on the appropriateness of altering the human genome for a particular purpose before any such research could proceed. The purpose of the proposed global moratorium was to provide time for careful study of the relevant technical and ethical issues to determine whether to pursue heritable human genome editing and, if that question were answered in the affirmative, to then determine how to proceed with making modifications to the human genome.

The whether of heritable human genome editing has not been resolved, and yet some scientists continue to race ahead with the how of it, essentially ignoring the myriad calls for public consultation. To be sure, other scientists are willing to heed the call, but would prefer to limit public consultation to public education.

I dont agree with this position. As I write in a new book, Altered Inheritance, we need to move the dial from public education (which typically is limited to talking at the public), to public engagement (which necessarily involves listening to the public), and then on to public empowerment (which is about shared decision-making).

To this end, we need slow science. Science needs time to think and to digest. Time is also needed to promote ethics literacy and to facilitate broad societal consensus where the goal is unity, not unanimity. Decision-making by consensus is about engaged, respectful dialogue and deliberation, where all participants recognize at the outset that knowledge is value laden; that we can and should learn from each other; and that no one should impose his or her will on others.

Read more: Could editing the DNA of embryos with CRISPR help save people who are already alive?

Metaphorically speaking, the human genome belongs to all of us. So we should all have a say in whether to proceed with making heritable changes to our shared genome. Decision-making by consensus, which begins with outreach and openness, is a means to this end. The goal is to create an environment in which all positions (not all persons) can be heard and understood, and in which there are reasonable opportunities for integrity-preserving compromises in pursuit of the common good. The underlying values are inclusivity, responsibility, self-discipline, respect, co-operation, struggle, and benevolence.

Scientists can meaningfully contribute to consensus building around genome editing. As individuals and as committee members, for example, they can effectively serve the common good by helping policymakers, legislators, and members of the public better align scientific information and opportunities with discrete values and interests.

I wrote Altered Inheritance as a call to action. It is a call for scientists to slow down, to reflect deeply on their science and their priorities, and to find meaningful ways to contribute to science policy in pursuit of the common good. It is also a call for all of us to take collective responsibility for the biological and social future of humankind as we think carefully about what kind of world we want to live in, and how genome editing technology might help us build that world.

Franoise Baylis is University Research Professor at Dalhousie University in Halifax, Nova Scotia, and author of Altered Inheritance: CRISPR and the Ethics of Human Genome Editing (Harvard University Press, September 2019).

Read more:
Opinion: Before heritable genome editing, we need slow science and dialogue within and across nations - Yahoo News

The Global CRISPR and Cas Genes Market Research Report Forecast 2019-2028: The research study has been prepared with the use of in-depth qualitative and quantitative analyses of the global CRISPR and Cas Genes Market. The report offers a complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the Global CRISPR and Cas Genes Market. It takes into account the CAGR, value, volume, revenue, production, consumption, sales, Manufacturing cost, prices, and other key factors related to the global CRISPR and Cas Genes Market.

The report helps the companies to better understand the CRISPR and Cas Genesmarket trends and to grasp opportunities and articulate critical business strategies. Also includes company profiles of market top companies like (contact information, product details, gross capacity, price, cost and more) are covered. this study of top companies in the market have been identified through secondary research, and their shares have been determined through primary and secondary research. and All percentage shares split, and breakdowns have been determined using secondary sources and verified primary sources.

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Key Players of the Global CRISPR and Cas Genes Market:

Addgene Inc, AstraZeneca Plc., Bio-Rad Laboratories Inc, Caribou Biosciences Inc, Cellectis S.A., Cibus Global Ltd, CRISPR Therapeutics AG, Editas Medicine Inc, eGenesis Bio, GE Healthcare, GenScript Corporation

Market Segmentation:

Segmentation on the basis of product:

Vector-based CasDNA-free CasSegmentation on the basis of application:

Genome EngineeringDisease ModelsFunctional GenomicsKnockdown/ActivationSegmentation on the basis of end user:

Biotechnology & Pharmaceutical CompaniesAcademic & Government Research InstitutesContract Research Organizations

Market Segment by Regions, regional analysis covers 2019-2028:

United States, Canada, and Mexico: North America

Germany, France, UK, Russia, and Italy: Europe

China, Japan, Korea, India, and Southeast Asia: Asia-Pacific

Brazil, Argentina, Colombia, etc.: South America

Saudi Arabia, UAE, Egypt, Nigeria, and South Africa: Middle East and Africa

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Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the global CRISPR and Cas Genes Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide CRISPR and Cas Genes Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the global CRISPR and Cas Genes Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the global CRISPR and Cas Genes Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global CRISPR and Cas Genes Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the global CRISPR and Cas Genes Market.

Market Forecast: Here, the report offers a complete forecast of the global CRISPR and Cas Genes Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

Appendix: Here, we have provided a disclaimer, our data sources, data triangulation, research programs, market breakdown and design, and our research approach.

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Global CRISPR and Cas Genes Market 2019 | Detailed Overview of the Market with Current and Future Industry Challenges and Opportunities - Stock Market...

DARPA Safe Genes concept

WASHINGTON: If you talk with senior defense officials there is one threat they will acknowledge is at the top of their list but they rarely discuss in public biological weapons. NowDARPA wants to develop defenses against biologically engineered threats before they are ever unleashed, the agencys director made clear this morning. That includes proactively editing troops DNA to produce a wide array of antibodies and biochemically blocking hostile attempts to edit DNA.

Our focus is about the protection aspect and the restoration, versus enhancement, Steven Walker said when I asked him about human augmentation during a CSIS conference. All these technologies, theyre dual use. You can use them for good; you can use them for evil and DARPA is about using them for good, to protect our warfighters.

Steven Walker

Super Immunity

That doesnt mean the US military has forsworn the genetic editing of human beings. To the contrary, Walker is very interested in ways to enhance the immune system, effectively turning the body into its own pharmaceutical factory.

Can you actually protect a soldier on the battlefield from chemical weapons and biological weapons by controlling their genome, [by] having the genome produce proteins that would protect the soldier from the inside out? he asked.

Well, why not just brew the necessary vaccines, anti-viral treatments, and anti-toxins in a normal factory and issue them as needed to the troops? Thats how weve dealt with naturally occurring diseases. And DARPA is working on that too, Walker said, with a program to build a vaccine in 60 days or less for 20,000 people for a virus youve never seen before.

The problem is that developing, producing, stockpiling, and dispensing one treatment at a time even in just 60 days may not work fast enough against future bioweapons. As soon as you develop a defense against one form of artificial plague, the enemy can use gene-editing tools to create a different version, one whose biochemical structure is just different enough that the old antibodies dont recognize it anymore.

Many diseases naturally mutate this way all the time. Thats why you can get a series of shots in childhood that protect you against measles or chicken pox for the rest of your life, but you need a new flu shot every year to stop the latest strain and no ones figured out how to stop the common cold. The so-called Spanish Flu of 1918-19 killed more people than World War I; imagine that as a weapon.

To have a shot available for every case that might be out there is becoming more and more intractable, because [of] synthetic biology and the ability of folks anywhere in the world to make something thats slightly different, Walker said. You cant stockpile enough of the vaccine or the antivirus capability to protect the population against that in the future.

Schematic of how CASPR Cas9 gene editing works

Undoing the Edit

DARPA is also looking at neutralizing or even reversing the effects of CRISPR Cas9 itself, the enzyme that made todays breakthroughs in gene-editing possible in the first place. (Its worth noting that China is now a leading country in gene editing science and its technology.)

How do we reverse it [genetic editing] if it gets out into the wild and gets out of control? Thats what the Safe Genes program is all about, Walker said. Weve actually made a lot of progress there in being able to control gene edits.

Walker didnt go into specifics, but theres plenty of non-military work in this area as well. Its even led by some of the pioneers of gene editing themselves, who understandably would like a way to undo the effects if one of their experiments goes wrong.

The irony of gene editing is that the crucial tool wasnt invented from scratch in the lab: It was found in nature. Many bacteria use CRISPR a whole complex of DNA sequences as a natural defense against invading viruses, allowing them to recognize the viral DNA as a foreign body and then use the Cas9 protein to cut it apart, killing the virus. (Though technically viruses arent living things in the first place). Scientists repurposed CRISPR Cas9 to snip apart and reorganize genes.

It turns out that, over millions of years of evolution, some viruses have developed an immunity to CRISP Cas9. They use so-called Anti-CRSPR proteins that shut down the enzyme so it cant start slicing DNA which would stop gene editing dead.

Lisa Porter

Benign Biotech

There are many more benign applications for biotechnology, said Walkers boss, Lisa Porter, the deputy under secretary of defense for research & engineering.

When we think biotech in DoD, we think chem/bio defense, and thats an element of the problem but theres also a lot of opportunity space that people dont necessarily realize unless they talk to the biologists, Porter told the CSIS conference. [So] we will be focusing, not just on the traditional tenets of biotech that we always do, but well be expanding into, what are the opportunities for new materials, new applications?

One biotech project she offered as an example is developing new materials to rapidly lay down new runways. Thats a matter of intense interest to the Air Force, which is increasingly worried its big central bases are easy targets for long-range missiles and wants new ways to either repair them or create alternative sites in a hurry.

What about human augmentation (boosted humans)? That gets a lot of concern and media attention, Porter said and quite rightly: You read about what China is doing and we should be concerned, because they dont have the same set of moral and ethical norms that we have in our country. (DARPA is careful to note in many of the web pages outlining genetic and related work that they work closely with recognized ethicists to ensure they are not crossing lines that should not be crossed.)

Porter, like Walker, did not mention any American plans to biologically enhance our own troops. But there are DARPA efforts that could, in the fast-changing world of biotech, lead to smarter, faster healing and stronger humans. Or try to stop what other countries have done to their troops.

Originally posted here:
DoD On Biotech: Build Sound Defenses First Breaking Defense - Defense industry news, analysis and commentary - Breaking Defense

Tracking nucleic acids in living cells

Fluorescence in situ hybridization (FISH) is a powerful molecular technique for detecting nucleic acids in cells. However, it requires cell fixation and denaturation. Wang et al. found that CRISPR-Cas9 protects guide RNAs from degradation in cells only when bound to target DNA. Taking advantage of this target-dependent stability switch, they developed a labeling technique, named CRISPR LiveFISH, to detect DNA and RNA using fluorophore-conjugated guide RNAs with Cas9 and Cas13, respectively. CRISPR LiveFISH improves the signal-to-noise ratio, is compatible with living cells, and allows tracking real-time dynamics of genome editing, chromosome translocation, and transcription.

Science, this issue p. 1301

We report a robust, versatile approach called CRISPR live-cell fluorescent in situ hybridization (LiveFISH) using fluorescent oligonucleotides for genome tracking in a broad range of cell types, including primary cells. An intrinsic stability switch of CRISPR guide RNAs enables LiveFISH to accurately detect chromosomal disorders such as Patau syndrome in prenatal amniotic fluid cells and track multiple loci in human T lymphocytes. In addition, LiveFISH tracks the real-time movement of DNA double-strand breaks induced by CRISPR-Cas9mediated editing and consequent chromosome translocations. Finally, by combining Cas9 and Cas13 systems, LiveFISH allows for simultaneous visualization of genomic DNA and RNA transcripts in living cells. The LiveFISH approach enables real-time live imaging of DNA and RNA during genome editing, transcription, and rearrangements in single cells.

Read the original:
CRISPR-mediated live imaging of genome editing and transcription - Science Magazine

The U.S. Patent and Trademark Office (USPTO) today awarded the University of California (UC), University of Vienna and Emmanuelle Charpentier a patent for CRISPR-Cas9 that, along with two others awarded this month, brings the teams comprehensive portfolio of gene-editing patents to 14.

Schematic representation of the CRISPR-Cas9 system. The Cas9 enzyme (orange) cuts the DNA (blue) in the location selected by the RNA (red). Image courtesy of Carlos Clarivan/Science Photo Library/NTB Scanpix

The newest patent, U.S. 10,415,061, covers compositions comprising single-molecule DNA-targeting RNAs or nucleic acids encoding single-molecule DNA-targeting RNAs, as well as methods of targeting and binding a target DNA, modifying a target DNA or modulating transcription from a target DNA with a complex that comprises a Cas9 protein and single-molecule DNA-targeting RNA.

On Sept. 10, the USPTO issued to the UC team U.S. patent 10,407,697 covering single-molecule guide RNAs or nucleic acid molecules encoding the guide RNAs. And on Sept. 3, the patent office issued U.S. patent 10,400,253, which covers compositions of single-molecule, DNA-targeting RNA (single-guide RNA, or sgRNA) and a Cas9 protein or nucleic acid encoding the Cas9 protein.

Another patent is set to issue next Tuesday, Sept. 24, bringing the total U.S. patent portfolio to 15. Three other patent applications have been allowed by the USPTO and are set to issue as patents in the coming months, which will raise the total to 18. These patents and applications span various compositions and methods for the CRISPR-Cas9 gene-editing technology, including targeting and editing genes and modulating transcription, and covering the technology in any setting, such as within plant, animal and human cells. The methods and compositions covered in UCs CRISPR-Cas9 portfolio come together to comprise the widest-ranging patent portfolio for the gene-editing technology.

This month, we have seen exponential growth of UCs U.S. CRISPR-Cas9 portfolio, said Eldora Ellison, Ph.D., lead patent strategist on CRISPR-Cas9 matters for UC and a director at Sterne, Kessler, Goldstein & Fox. We remain committed to expanding our robust portfolio to include additional methods and compositions for CRISPR-Cas9 gene editing so that the range of applications can be fully utilized for the benefit of humanity.

The team that invented the CRISPR-Cas9 DNA-targeting technology included Doudna and Martin Jinek at UC Berkeley; Charpentier, then at Umea University in Sweden and now director of the Max Planck Institute for Infection Biology in Germany; and Krzysztof Chylinski of the University of Vienna. The methods covered by todays patent, as well as the other methods claimed in UCs previously issued patents and those set to issue, were included among the CRISPR-Cas9 gene editing technology work disclosed first by the Doudna-Charpentier team in its May 25, 2012, priority patent application.

The 14 CRISPR-Cas9 patents in this teams portfolio are 10,000,772; 10,113,167; 10,227,611; 10,266,850; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,358,658; 10,358,659; 10,385,360; 10,400,253; 10,407,697; and 10,415,061. These patents are not a part of the PTABs recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broads CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 14 patents granted in the U.S. so far. The European Patent Office (representing more than 30 countries), as well as patent offices in the United Kingdom, China, Japan, Australia, New Zealand, Mexico, and other countries, have issued patents for the use of CRISPR-Cas9 gene editing in all types of cells.

University of California has a long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind. Consistent with its open-licensing policies, UC allows nonprofit institutions, including academic institutions, to use the technology for non-commercial educational and research purposes.

In the case of CRISPR-Cas9, UC has also encouraged widespread commercialization of the technology through its exclusive license with Caribou Biosciences, Inc. of Berkeley, California. Caribou has sublicensed this patent family to numerous companies worldwide, including Intellia Therapeutics, Inc. for certain human therapeutic applications. Additionally, Dr. Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Limited.

Go here to read the rest:
CRISPR portfolio now at 14 and counting - UC Berkeley

Its been an alarming year for the worlds outlook on biodiversity. The Intergovernmental Science-Policy Platform on Biodiversity and Ecosystem Services (IPBES) put the world on notice that around 1 million species are facing extinction (PDF). A study published in August concluded that it would take New Zealand 50 million years to recover the diversity of bird species it has lost since human colonization. And, while headlines about an insect apocalypse may have been hyperbolic, insect biodiversity is decreasing, and its a problem.

As evidenced in the IPBES report, current conservation efforts have not been sufficient to stem biodiversity loss, so innovative solutions might be necessary to support the web of life that supports human existence. In 2012, scientists first described the gene editing capabilities of CRISPR, a molecular tool that can be used to make targeted, precise changes to the DNA of plants, animals and microbes.

Since then, scientists have proposed myriad ways to use the technology. But could it be a boon to biodiversity? Can it help researchers understand and preserve corals and their ecosystems? What about applications to diversify agriculture to shore up food security? Or to combat invasive species plaguing ecosystems around the world?

While many scientists are eager to discuss the possibilities of using CRISPR to preserve biodiversity, they are also cautious. The effects of human interventions are not always predictable, and once a gene-edited species is released into the wild, controlling any negative effects will be difficult. Toni Piaggio, a research scientist at the U.S. Department of Agriculture (USDA) National Wildlife Research Center, says researchers should "never entirely sip the Kool-Aid" when it comes to CRISPR. Instead, she says, they should "spend a lot of research time and intellectual energy" questioning themselves and their work.

While many scientists are eager to discuss the possibilities of using CRISPR to preserve biodiversity, they are also cautious.

Diversity for food security

But as millennia passed, domestication also decreased the genetic diversity within the plants we grow and eat. To understand why, imagine an ancient human 10,000 years ago, tired of smashing teosinte with rocks to get a few measly kernels out of their hard casings. If that person saw a plant with naked kernels exposed and available to eat without rock smashing they might select seeds from that plant to grow the next year. That works out great for the person, but the genetic diversity in the rest of the field is lost to future generations.

The same forces are at play today. When each tomato plant, for example, looks the same, grows at the same rate and produces pounds upon pounds of tomatoes, farming is easier and the food supply is more predictable if everything goes as usual.

Problem is, farming doesnt always follow usual, expected patterns. And climate change is increasing variability and unpredictability in agriculture. Many crops, as a result of their low genetic diversity, are not particularly well suited to cope with emerging climate patterns, leaving them susceptible to challenges such as drought, flooding or salty soils. So, says Lzaro Peres, a professor of plant physiology at the University of So Paulo, relying on a limited number of crop species to produce the worlds food is risky.

Peres and other researchers are trying to infuse agriculture with the genetic diversity of wild species. His research team started with a wild tomato and used CRISPR to edit a handful of key genes. Its goal was to make the versions of the genes in wild tomato look like the versions of the genes in domesticated tomato. In doing so, the wild tomato species gained some beneficial characteristics common to domesticated species. Through this process, de novo domestication, Peres and colleagues produced a tomato with more fruit, bigger fruit and more lycopene than wild tomatoes and that are genetically diverse from conventional domesticated tomatoes.

Does a change in plant size or color affect which insects are attracted to it? How does that affect the predators of those insects?

But, looking beyond a single crop into the ecosystem within which it exists is important, says Yolanda Chen, an associate professor in the College of Agriculture and Life Sciences at the University of Vermont. Chen studies the impact plant domestication can have on insect populations. She says that researchers need to consider how genes "operate within a broader community context" and not just in a single plant. Does a change in plant size or color affect which insects are attracted to it? How does that affect the predators of those insects?

Peres is mindful of the potential effects on agricultural ecosystems. Domesticating a wild tomato and growing it at scale could impact nuanced ecological relationships. Still, he says, he "sees mainly positive things" about the potential impacts of his work. "And one of the things is food security, because it is quite dangerous to depend on very few species for our food, feed and fiber."

Chen says that she thinks gene editing for de novo domestication is "less risky" than other genetic approaches, such as those that introduce entire new genes into a plant species. In de novo domestication, the edited versions of genes already exist in related domesticated tomato plants.

It likely will be a while before a new species of tomato developed to increase the genetic diversity of our food is available at the local grocery store. Peres says the work he and his group have published so far was a proof of concept; in other words, they showed that de novo domestication is feasible, but have no plan to commercialize that tomato. Theyve since turned their attention to a species of wild tomato from the Galpagos Islands that grows especially well in salty soils and is resistant to a white fly that can cause severe crop damage. If they are able to de novo domesticate this tomato, it could be used as an important crop for farmers dealing with salty, coastal soils.

In the end, Chen and Peres are both concerned about climate change, agriculture and biodiversity. They approach solutions to these concerns from different research perspectives, but both see diversity on the genetic and species levels in agricultural ecosystems as an important aspect of a food system that can withstand the challenges of climate change. In the future, domesticating new plant species potentially with gene editing might give farmers more options for growing diverse crops well-suited to specific climates.

Coral conservation

In 1770, British explorer Captain James Cook ran his ship, Endeavor, aground on the "insane labyrinth" that would become known as the Great Barrier Reef off the coast of Queensland, Australia. While Cook was credited with "discovering" the reef, coral reefs had been important to indigenous people for centuries before.

A few hundred years later, pollution and warming water have resulted in huge coral bleaching events around the world. While corals can survive bleaching, the stress does lead to increased mortality. Thats bad news for the marine species that inhabit corals. When corals are lost, reef ecosystems suffer, throwing the relationships between the thousands of species including fish, invertebrates, plants and turtles that live there out of balance.

Current conservation efforts for the worlds corals have been insufficient to curb bleaching events and sustain the valuable ecosystems corals support, according to the IPBES report. So there is a certain urgency to finding new approaches to conservation. A 2019 report by biologists laid out different conservation approaches and evaluated their potential risks and benefits. And with the 2018 announcement that scientists have used CRISPR to edit genes in coral, gene editing is seen as a potential strategy. Maybe.

Current conservation efforts for the worlds corals have been insufficient to curb bleaching events and sustain the valuable ecosystems corals support.

Marie Strader, now an assistant research professor at Auburn University, was a lead researcher as a graduate student on the international team of scientists that produced the work. The scientists edited three types of genes in a vibrantly colored coral called Acropora millepora. The goal of the editing was to "break" or mutate the genes, and in some larvae, it did.

As this proof-of-concept study was successful meaning they were able to edit the coral genes they targeted at least some of the time other researchers can use their methods as a blueprint for editing other genes in Acropora millepora and editing other coral species. For starters, Strader says, theyll likely look at genes involved in the coral life cycle and temperature sensitivity. Understanding those processes, Strader says, can "translate into conservation efforts down the line."

For example, researchers can use CRISPR in the lab to help them understand which genes are important for tolerance to warm waters. If they edit a gene in the lab and the resulting coral can better tolerate warm waters, according to Strader, the scientists could look at natural coral populations for those that naturally have that genetic mutation. Armed with that understanding, researchers might be more successful at conservation efforts such as breeding corals to help them keep their cool as the heat turns up.

If they edit a gene and the resulting coral can better tolerate warm waters, the scientists could look at natural coral populations for those that naturally have that genetic mutation.

For one thing, there are still plenty of technical obstacles. In Straders work, individual edited corals ended up with a mix of edited and unedited copies of the genes. To realize the full effect of a gene edit and to pass it down to future generations, each cell of the coral ideally should have the same edit. And other details, such as making sure CRISPR edits only the targeted gene or genes, "need to be worked out before it would be a viable option for conservation purposes," Strader says.

Furthermore, says John Bruno, a marine ecologist at the University of North Carolina at Chapel Hill, conservation efforts need to protect not just corals but also the thousands of other species that rely on them. According to Bruno, gene editing 10 or 20 species of corals to tolerate warm water just isnt enough. As "nobodys going to CRISPR all billion species that are in the ocean," he says, conservation needs to focus on the whole ecosystem and not just a few species. "The solution is rather obvious, just radically mitigate greenhouse gas emissions," he says acknowledging thats no easy feat.

Running interference

The situation with corals is "dire," according to Bruno. But even in coral species that have seen precipitous declines, often still many potentially on the order of millions of individuals are left, he says.

Back on shore, some animal populations are much smaller and easily could slip out of existence under the thumb of invasive species. In New Zealand, native birds evolved without mammalian predators. Many are large and flightless, so when mammals such as rats, possums and stoats arrived with humans, the birds were easy targets. According to one study, these invasive animals are responsible for the loss of an estimated 26.6 million chicks and eggs of native bird species each year.

Gene drives, which have become more plausible with the advent of gene editing, could offer a more humane way of managing invasive populations and protecting the species they endanger.

Gene drives, which have become more plausible with the advent of gene editing, could offer a more humane way of managing invasive populations and protecting the species they endanger.

"So many things have been done with the best possible intention, and we find that theres just been unforeseen consequences," says Helen Taylor, a conservation geneticist and honorary research fellow at the University of Otago. She points out that while possums are pests in New Zealand, they are an important species in Australia. If a possum with the New Zealand gene drive somehow were released in Australia, the effects could be devastating.

Maud Quinzin, a conservation geneticist and senior postdoctoral associate, recently began working in MITs Sculpting Evolution Lab with Kevin Esvelt, the scientist who first proposed CRISPR as a tool to create gene drives. Quinzin is using her understanding of ecosystem dynamics to help the Sculpting Evolution Lab think about the complex rippling effects of human interference in ecosystems.

Its important to look at the science from all angles, she says. "Developing gene-editing tools requires scientists with very different expertise sharing ideas and progress from early on in the process." For example, if an invasive rat species is eradicated from an island, will other species even other invasive species become more populous? "You have to think about the dynamic in that ecosystem," she says. Since suggesting that CRISPR could be used for gene drives, Esvelt himself has been vocal about his concerns.

Still, Quinzin has been on the front lines of conservation biology, watching populations of valued species go extinct, and shewants communities to be presented with all options for conservation. For scientists to present those options, though, they really need to understand the places where they might work, Quinzin says. That understanding comes not just from researchers, but also from the people who live in those places. "It is really important that you respect the values and the knowledge in a place," Quinzin says, including "not only the scientific information but also the indigenous or local knowledge." By engaging with local communities as technology develops, Quinzin says, researchers can focus on developing technology in ways that align with a communitys cultural, social, political and environmental values.

Moving forward

In the short term, agriculture might be the most likely use of CRISPR to protect biodiversity. In fact, the first gene-edited crop hit the market in the United States in early 2019. Individual countries are still figuring out how to regulate edited plants, with a big distinction being made between plants that could have emerged through natural mutations and plants containing larger edits, such as those containing new DNA.

At the very least, the work of scientists such as Peres could expand the genetic diversity of our crop plants, adding more options to the table as farmers, scientists and other stakeholders work toward a food-secure world. And having options is important. No single solution can save biodiversity everywhere. And carelessly applied solutions can cause more problems.

Scientists do seem to be proceeding with caution. At least some coral researchers decline to consider using CRISPR in the wild. Scientists studying gene drives are vocally pointing out the limitations of the technology and extolling the role nonscientists must play in the decisions to use or not use CRISPR for conservation purposes.

"I think we have a really big not just opportunity, but an obligation to get it out there in the public eye as much as possible," Piaggio says. And if scientists dont get public buy-in, they shouldnt use the technology, she says. "I think we have to be OK with that."

Quinzin says that she and other scientists in her group want guidance from the public. At the same time, she notes that CRISPR "could be such an amazing tool if we are respectful [and] responsible and use it properly."

There are no perfect or universal solutions to the biodiversity crisis the world is facing. And the causes cannot be forgotten in pursuit of an antidote. Thats why it will take scientists and conservationists with diverse approaches working in different areas to make a difference.

This article was originally published on Ensia.

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Can the gene editing technology CRISPR help reduce biodiversity loss worldwide? - GreenBiz

A gene-editing technique that has shown promise as a potential cure for sickle-cell disease is now being tested in humans. But if it works, will the people who need it even be able to get it? Now that a cure may be in sight, this is an urgent question, says Vence Bonham, a senior advisor to the director of the National Human Genome Research Institute.

Sickle-cell disease (SCD) is a genetic blood disorder that affects millions of people in the world. It causes the production of abnormal red blood cells and can lead to intense pain, strokes, and organ and tissue damage.

From a scientific perspective, its an exciting time for people who suffer from the disease, Bonham said today at MIT Technology Reviews EmTech conference. Researchers are testing a technique that uses the precise gene-editing tool CRISPR to modify a single gene associated with the disease.

Justin Saglio

But from a sociological standpoint, argued Bonham, the work is just beginning. SCD is more common in certain ethnic groups, particularly people of African descent. And while there are around 100,000 people with the disease in the US, the vast majority live in sub-Saharan Africa and India, Bonham said.

He and colleagues recently conducted a study intended to explore the attitudes and beliefs toward the promising technique held among people with SCD, their family members, and their physicians. Many of the people Bonham and his colleagues spoke with expressed skepticism that a potential CRISPR-based cure would be affordable and accessible to those who need it. Although they did find renewed hope, they also observed cautionary, apprehensive undertones to this hope, which they concluded stem in part from decades of medical disenfranchisement of the SCD community.

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According to one physician interviewed for the study, there is a danger that other rare diseases that tend to affect people with more resources might get more attention and, potentially, funding. As a result, there is a concern that the SCD population could get left in the dust. This population is already skeptical, since they have been left in the dust with so many other things, the physician added.

Besides a cure itself, we also need better and cheaper ways to expand the benefits of this new technology, Bonham said. The potential is great, but we must ask the question: Who will benefit?

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CRISPR could help us cure sickle-cell disease. But patients are wary. - MIT Technology Review

(more about Power Words)

cancerAny of more than 100 different diseases, each characterized by the rapid, uncontrolled growth of abnormal cells. The development and growth of cancers, also known as malignancies, can lead to tumors, pain and death.

Cas9An enzyme that geneticists are now using to help edit genes.It can cut through DNA, allowing it to fix broken genes, splice in new ones or disable certain genes. Cas9 is shepherded to the place it is supposed to make cuts by CRISPRs, a type of genetic guides. The Cas9 enzyme came from bacteria. When viruses invade a bacterium, this enzyme can chop up the germs DNA, making it harmless.

cellThe smallest structural and functional unit of an organism. Typically too small to see with the unaided eye, it consists of a watery fluid surrounded by a membrane or wall. asyeasts, molds, bacteria and some algae, are composed of only one cell.

clinicaltrialA research trial that involves people.

CRISPRAn abbreviation pronounced crisper for the term clustered regularly interspaced short palindromic repeats. These are pieces of RNA, an information-carrying molecule. They are copied from the genetic material of viruses that infect bacteria. When a bacterium encounters a virus that it was previously exposed to, it produces an RNA copy of the CRISPR that contains that virus genetic information. The RNA then guides an enzyme, called Cas9, to cut up the virus and make it harmless. Scientists are now building their own versions of CRISPR RNAs. These lab-made RNAs guide the enzyme to cut specific genes in other organisms. Scientists use them, like a genetic scissors, to edit or alter specific genes so that they can then study how the gene works, repair damage to broken genes, insert new genes or disable harmful ones.

disorder(in medicine) A condition where the body does not work appropriately, leading to what might be viewed as an illness. This term can sometimes be used interchangeably with disease.

DNA(short for deoxyribonucleic acid) Along, double-stranded and spiral-shaped molecule inside most living cells that carries genetic instructions. It is built on a backbone of phosphorus, oxygen, and carbon atoms. In all living things, from plants and animals to microbes, these instructions tell cells which molecules to make.

engineerA person who uses science to solve problems. As a verb, to engineer means to design a device, material or process that will solve some problem or unmet need.

gene(adj. genetic) A segment of DNA that codes, or holds instructions, for a cells production of a protein. Offspring inherit genes from their parents. Genes influence how an organism looks and behaves.

geneticHaving to do with chromosomes, DNA and the genes contained within DNA. The field of science dealing with these biological instructions is known as genetics. People who work in this field are geneticists.

hemoglobinA molecule that binds to oxygen in the blood, carrying it around to tissues.

immune(adj.) Having to do with the immunity. (v.) Able to ward off a particular infection.Alternatively, this term can be used to mean an organism shows no impacts from exposure to a particular poison or process. More generally, the term may signal that something cannot be hurt by a particular drug, disease or chemical.

insightThe ability to gain an accurate and deep understanding of a situation just by thinking about it, instead of working out a solution through experimentation.

multiplemyelomaThis cancer starts in a type of white blood cells known as plasma cells. Part of the immune system, they help guard the body from germs and other harmful substances.

muscleA type of tissue used to produce movement by contracting its cells, known as muscle fibers. Muscle is rich in protein, which is why predatory species seek prey containinglots of this tissue.

mutation(v. mutate) Some change that occurs to a gene in an organisms DNA. Some mutations occur naturally. Others can be triggered by outside factors, such as pollution, radiation, medicines or something in the diet. A gene with this change is referred to as a mutant.

nerveA long, delicate fiberthat transmits signalsacross the body of an animal. An animals backbone contains many nerves, some of which control the movement of its legs or fins, and some of which convey sensations such as hot, cold or pain.

neuronAn impulse-conducting cell. Such cells are found in the brain, spinal column and nervous system.

oxygenA gas that makes up about 21 percent of Earth's atmosphere. All animals and many microorganisms need oxygen to fuel their growth (and metabolism).

pharmaceuticalsMedicines, especially prescription drugs.

plasma (in medicine) The colorless fluid part of blood.

proteinA compoundmade from one or more long chains of amino acids. Proteins are an essential part of all living organisms. They form the basis of living cells, muscle and tissues; they also do the work inside of cells. Among the better-known, stand-alone proteins are thehemoglobin (in blood) and the antibodies (also in blood) that attempt to fight infections. Medicines frequently work by latching onto proteins.

redblood cellColored red by hemoglobin, these cells move oxygen from the lungs to all tissues of the body. Red blood cells are too small to be seen by the unaided eye.

retinaA layer at the back of the eyeball containing cells that are sensitive to light and that trigger nerve impulses that travel along the optic nerve to the brain, where a visual image is formed.

RNAA molecule that helps read the genetic information contained in DNA. A cells molecular machinery reads DNA to create RNA, and then reads RNA to create proteins.

sarcomaA family of more than 70 cancers that begin in bones or in connective tissues.

technologyThe application of scientific knowledge for practical purposes, especially in industry or the devices, processes and systems that result from those efforts.

therapy(adj. therapeutic) Treatment intended to relieve or heal a disorder.

variantA version of something that may come in different forms. (ingenetics) A gene having a slight mutation that may have left its host species somewhat better adapted for its environment.

wombAnother name for the uterus, the organ in mammals in which a fetus grows and matures in preparation for birth.

Original post:
Genetics CRISPR enters its first human trials - Science News for Students

In a recent review, scientists highlight the potential of gene editing technologies like CRISPR/Cas9 to not only understand the molecular mechanisms behind Parkinsons disease, but also identify new targets for treatment.

The review study, Interrogating Parkinsons disease associated redox targets: Potential application of CRISPR editing, was published in the journal Free Radical Biology and Medicine.

One of the hallmarks of PD is the loss of dopamine-producing neurons in the substantia nigra a brain region involved in the control of voluntary movements, and one of the most affected in PD. This occurs due to the clustering of a protein called alpha-synuclein in structures commonly known as Lewy bodies inside neurons.

Parkinsons is complex and multifactorial disease, with both genetic and environmental factors playing a role in either triggering or exacerbating the disease.

Genetic causes can explain 10% of all cases of PD called familial PD , meaning that in the majority of the cases (sporadic PD) there is an interplay between genetics and environmental risk factors.

Researchers atSechenov Universityin Russia and theUniversity of Pittsburgh reviewed the role of metabolic pathways, especially problems with mitochondria cells powerhouses and iron accumulation, as well as mechanisms in cell death (called apoptosis and ferroptosis) in the development and progression of Parkinsons disease.

These processes were discussed in the context of genome editing technologies, namely CRISPR/Cas9 a technique that allows scientists to edit genomes, inserting or deleting DNA sequences, with precision, efficiency and flexibility.

CRISPR is a promising technology, a strategy to find new effective treatments to neurodegenerative diseases, Margarita Artyukhova, a student at the Institute for Regenerative Medicineat Sechenov and the study first author, said in a press release.

Mitochondria dont work as they should in people withPD, resulting in shortages of cellular energy that cause neurons to fail and ultimately die, particularlydopamine-producing neurons. Faulty mitochondria are also linked to the abnormal production of reactive oxygen species, leading to oxidative stressan imbalance between the production of free radicals and the ability of cells to detoxify them that also damages cells over time.

Because mitochondrial dysfunction is harmful, damaged mitochondria are usually eliminated (literally, consumed and expelled) in a process called mitophagy an important cleansing process in which two genes, called PINK1 and PRKN, play crucialroles. Harmful changes in mitophagy regulation is linked with neurodegeneration in Parkinsons.

Previous studies with animal models carrying mutations in the PINK1and PRKNgenes showed that these animals developed typical features of PD mitochondrial dysfunction, muscle degeneration, and a marked loss of dopamine-producing neurons.

PINK1codes for an enzyme that protects brain cells against oxidative stress, whilePRKNcodes for a protein called parkin. Both are essential for proper mitochondrial function and recycling by mitophagy. Mutations in both the PINK1 and PRKNgene have been linked with early-onset PD.

However, new research suggests that the role of PINK1 and PRKNin Parkinsons could be more complex and involve other genes likePARK7(DJ-1), SNCA (alpha-synuclein) andFBXO7 as well as a fat molecule called cardiolipin.

CRISPR/Cas9 genome editing technology may be used to help assess the role of different genetic players in Parkinsons disease, and to look for unknown genes associated with disease progression and development. Moreover, this technology can help generate animal and cellular models that might help scientists decipher the role of certain proteins in Parkinsons and discover potential new treatment targets.

Iron is another important metabolic cue in Parkinsons. While its essential for normal physiological functions, excessive levels of iron can be toxic and lead to the death of dopamine-producing neurons in the substantia nigra.

Iron may also interact with dopamine, promoting the production of toxic molecules that damage mitochondria and cause alpha-synuclein buildup within neurons.

CRISPR/Cas9 technology can be used to help dissect the role of proteins involved in iron transport inside neurons, which in turn may aid in designing therapies to restore iron levels to normal in the context of Parkinsons disease.

Finally, researchers summarized evidence related to the role of two cell death pathways ferroptosis and apoptosis in PD. Ferroptosis is an iron-dependent cell death mechanism by which iron changes fat (lipid) molecules, turning them toxic to neurons. This process has been implicated in cell death associated with degenerative diseases like Parkinsons, and drugs that work to inhibit ferroptosis have shown an ability to halt neurodegeneration in animal models of the disease.

Apoptosis refers to a programmed cell death mechanism, as opposed to cell death caused by injury. Both apoptosis and ferroptosis speed the death of dopaminergic neurons.

CRISPR/Cas9 may help to pinpoint the key players in cell death that promote the loss of dopaminergic neurons in Parkinsons disease, while understanding the array of proteins that are involved in these processes.

These insights into the mechanisms of PD pathology [disease mechanisms] may be used for the identification of new targets for therapeutic interventions and innovative approaches to genome editing, including CRISPR/Cas9, the researchers wrote.

Genome editing technology is currently being used in clinical trials to treat patients with late-stage cancers and inherited blood disorders, Artyukhova notes in the release.

These studies allow us to see vast potential of genome editing as a therapeutic strategy. Its hard not to be thrilled and excited when you understand that progress of genome editing technologies can completely change our understanding of treatment of Parkinsons disease and other neurodegenerative disorders, she adds.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

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CRISPR/Cas9 Potential in Advancing Parkinson's Understanding and Treatment Focus of Review Study - Parkinson's News Today

We are pleased to announce Technology Networks Explores the CRISPR Revolution. Through a series of exclusive interviews with world-renowned scientists and bioethicists, Technology Networks Explores the CRISPR Revolution will investigate the ground-breaking research taking place in the CRISPR space, CRISPR "controversies" and whether the CRISPR technology looks set to fulfil its promise of revolutionizing science.

The series will feature interviews with researchers behind the discovery of the CRISPR mechanism, such as Professor Francisco Mojica, the scientist involved its development as a gene-editing tool, Professor Jennifer Doudna, and the "godfather" of human genome research, Professor George Church.

The series will also explore the latest technologies available in the CRISPR "toolbox" including industry perspectives, its application in agriculture and farming through a conversation with Professor Alison Van Eenennaam and insights into the global conversation surrounding its ethical implications from Professor Glenn Cohen.

Kicking off the series on Oct 14th is an interview with the humble and immensely influential microbiologist, Professor Francisco Mojica.

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Technology Networks Explores the CRISPR Revolution Coming Soon - Technology Networks

Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers at the University of Missouri School of Medicine have shown in a mouse study that the powerful gene editing technique known as CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder.

Children with DMD have a gene mutation that interrupts the production of a protein known as dystrophin. Without dystrophin, muscle cells become weaker and eventually die. Many children lose the ability to walk, and muscles essential for breathing and heart function ultimately stop working.

"Research has shown that CRISPR can be used to edit out the mutation that causes the early death of muscle cells in an animal model," said Dongsheng Duan, PhD, Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and the senior author of the study. "However, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells."

In collaboration with other MU colleagues and researchers from the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, Duan explored whether muscle stem cells from mice could be efficiently edited. The researchers first delivered the gene editing tools to normal mouse muscle through AAV9, a virus that was recently approved by the U.S. Food and Drug Administration to treat spinal muscular atrophy.

"We transplanted AAV9 treated muscle into an immune-deficient mouse," said Michael Nance, a MD-PhD program student in Duan's lab and the lead author of the paper. "The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene."

The researchers' reasoning was correct, as they found abundant edited cells in the regenerated muscle. They then tested if muscle stem cells in a mouse model of DMD could be edited with CRISPR. Similar to what they found in normal muscle, the stem cells in the diseased muscle were also edited. Cells regenerated from these edited cells successfully produced dystrophin.

"This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases," Duan said. "Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout a patient's life."

With more study, the researchers hope this stem cell-targeted CRISPR approach may one day lead to long-lasting therapies for children with DMD.

Reference: Nance et al. 2019.AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice. Molecular Therapy.DOI: https://doi.org/10.1016/j.ymthe.2019.06.012.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Researchers Use CRISPR to Correct Mutation in Duchenne Muscular Dystrophy Model - Technology Networks

A team has used a lentiviral capsid-based bionanoparticle system to deliver CRISPR-Cas9 gene editing therapies, reducing undesired effects.

Researchers have developed an improved CRISPR delivery system for gene editing, through a lentiviral capsid system. The team say that their findings could be useful in research and clinical applications by improving safety and avoiding possible immune responses.

using a traditional lentiviral vector allows the bionanoparticle to efficiently and safely deliver CRISPR-Cas9

The team, from Wake Forest Institute of Regenerative Medicine (WFIRM), US, packaged the Cas9 protein and guide RNA together within a lentiviral capsid-based bionanoparticle system.

Previously, the two components had to be delivered separately which was not as convenient, said Dr Baisong Lu, assistant professor of regenerative medicine at WFIRM and one of the lead authors of the paper.

Conventional CRISPR-Cas9 is not completely accurate and could potentially cut unexpected locations, causing unwanted results.

However, the using a traditional lentiviral vector allows the bionanoparticle to efficiently and safely deliver CRISPR-Cas9. The researchers observed that it reduced off-target rates compared to regular CRISPR-Cas9.

A similar strategy should be translatable to other editor proteins for gene disruption, said Anthony Atala, MD, director of WFIRM and a co-author of the paper. We may be able to utilise this to package and deliver other RNPs into mammalian cells, which has been difficult to achieve so far.

The findings were published in Nucleic Acids Research.

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Researchers improve CRISPR-Cas9 delivery efficiency - Drug Target Review

UK-based Oxford Nanopore has obtained a licence to CRISPR-Cas9 IP for nanopore sequencing, a third-generation approach used in the sequencing of biopolymers.

Oxford Nanopore, which specialises in DNA/RNA sequencing technology, announced the non-exclusive licence agreement with biotech company Caribou Biosciences yesterday, September 19.

Caribou was founded by scientists from the University of California, Berkeley, including CRISPR pioneer Jennifer Doudna.

Gordon Sanghera, CEO of Oxford Nanopore, said: The Cas9 technique will enable users to select and isolate the regions of the genome they are most interested in, including those not available to existing methods, ready for rapid analysis using our long-read, real-time sequencing technology.

According to the company, Cas9 enrichment with Oxford Nanopore sequencing enables scientists to cost-effectively sequence targeted regions that were not accessible previously.

Sanghera added: The entire library preparation process takes less than two hours so if combined with our portable sequencer MinION, this has the potential to open up fast-turnaround, near-sample testing in new ways.

In October last year, Amgen invested 50 million ($66 million) in Oxford Nanopore, as part of Amgens focus on using human genetics to deliver new medicines to patients.

Earlier in 2018, Oxford Nanopore announced it had raised 100 million from global investors, to be used to support the companys next phase of commercial expansion, including a new high-tech manufacturing facility in Oxford.

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Oxford Nanopore, CRISPR-Cas9, Caribou Biosciences, Jennifer Doudna, gene-editing, genetics, nanopore, University of California,

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Oxford Nanopore signs CRISPR licence - Life Sciences Intellectual Property Review

At this time, there is no cure for Duchenne muscular dystrophy (DMD), although there is one treatment for a subgroup of the disease. That is Sarepta Therapeutics Exondys 51 for DMD patients with a confirmed mutation amenable to exon 51 skipping. Recently the U.S. Food and Drug Administration (FDA) rejected Sareptas golodirsen for DMD with a confirmed mutation appropriate for exon 53 skipping.

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.

There just might be, however, hope for an actual cure. Researchers at the University of Missouri-Columbia, utilized CRISPR gene editing in a mouse model, to edit out the gene mutation and transplant AAV9 treated muscle into the mice. The transplanted muscle cells carried the edited gene and successfully produced dystrophin, the protein that is not produced in sufficient quantities in DMD patients.

The dystrophin gene is the largest in the body, and codes for the dystrophin protein, which is involved in muscle development and activity. One of the reasons DMD has been a tough nut to crack is that because of the genes size, its too large to fit into the typical viral vectors used in gene therapies. Thats partially why Sareptas approach is to use a type of RNA splicing that forces cells to skip over the faulty section of genetic code. This results in a shortened (truncated) protein that is still functional.

Research has shown that CRISPR can be used to edit out the nutation that causes the early death of muscle cells in an animal model, said Dongsheng Duan, the Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine and senior author of the study.

However, Duan went on, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells.

Duans research, in collaboration with others at MU as well as the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, looked at whether muscle stem cells in mice could be effectively edited. They used AAV9, an adeno-associated virus recently approved by the FDA to treat spinal muscular atrophy (SMA)Novartis Zolgensma, which is also the source of the controversy over the companys data manipulation scandal.

They started by delivering CRISPR to normal mouse muscle via AAV9.

We transplanted AAV9-treated muscle into an immune-deficient mouse, said Michael Nance, an MD-PhD program student in Duans lab and the lead author of the paper. The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene.

That appeared to work. They then tested if the muscle stem cells in the mice of DMD could be edited with CRISPRthey were.

This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases, Duan said. Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout the patients life.

The research was published in the journal Molecular Therapy.

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CRISPR Research Might Lead to Cure for Duchenne Muscular Dystrophy - BioSpace