Archive for the ‘Crispr’ Category

CrisprCon is not a place where spandexed, beglittered, refrigerator drawer fans come together for an all-you-can-eat celebration of unwilted produce. No. Crispr-Cas9 (no E), if you havent been paying attention, is a precise gene editing tool thats taken the world by storm, promising everything from healthier, hangover-free wine to cures for genetic diseases. Like, all of them. And CrisprCon is where people come not to ask how to do those things, but rather, should we? And also, whos the we here?

On Wednesday and Thursday, the University of California, Berkeley welcomed about 300 peoplescientists, CEOs, farmers, regulators, conservationists, and interested citizensto its campus to take a hard look at the wnderenzyme known as Cas9. They discussed their greatest hopes and fears for the technology. There were no posters, no p-values; just a lot of real talk. You can bet it was the first Crispr conference to sandwich a Cargill executive between a septagenarian organic farmer and an environmental justice warrior. But the clashing views were a feature, not a bug. "When you feel yourself tightening up, that's when you're about to learn something," said moderator and Grist reporter, Nathanael Johnson.

Which, to be honest, was totally refreshing. Serious conversations about who should get to do what with Crispr have been largely confined to ivory towers and federal agencies. In February the National Academy of Sciences released a report with its first real guidelines for Crispr, and while it suggested limitations on certain applicationslike germline modificationsit was largely silent on questions outside of scientific research. What sorts of economies will Crispr create; which ones will it destroy? What are the risks of using Crispr to save species that will otherwise go extinct? Who gets to decide if its worth it? And how important is it ensure everyone has equal access to the technology? Getting a diverse set of viewpoints on these questions was the explicit goal of CrisprCon

Why was that important? Greg Simon, director of the Biden Cancer Initiative and the conferences keynote speaker, perhaps said it best: Crispr is not a light on the nation, its a mirror. In other words, its just another technology thats only as good as the people using it.

Panel after panel took the stage (each one, notably, populated with women and people of color) and discussed how other then-cutting-edge technologies had failed in the past, and what history lessons Crispr users should not forget. In the field of conservation, one panel discussed, ecologists failed to see the ecosystem-wide effects of introduced species. As a result, cane toads, red foxes, and Asian carp created chaos in Australia and New Zealand. How do you prevent gene drivesa technique to spread a gene quickly through a wild populationfrom running similarly amok?

From the agricultural field, the lessons were less nebulous. First-generation genetically modified organisms failed to gain public support, said organic farmer Tom Willey, because they never moved agriculture in a more ecologically sustainable direction and it never enhanced the quality of food people actually ate. At least, noticeably so. Instead, most modifications were to commodity crops like corn and soy to improve their pest resistance or boost yields.] It was a convenience item for farmers, he said. And a profit center for corporations. In order for gene-edited foods to avoid the same fate, companies like Monsanto, Dupont Pioneer, and Cargill, who have already licensed Crispr technologies, will need to provide a more tangible value than corn you can spray the bejeezus out of. Like say, extra-nutritious tomatoes, or a wine with 10-times more heart-healthy resveratrol and fewer of the hangover-causing toxins.

The presence of executives from each of these three companies signaled that theyre serious about not making the same mistakes they did in the 90s when GMOs first came to market. Back then we were only talking to farmers, said Neal Gutterson, vice president of R&D at Dupont Pioneer during a break between panels. I cant remember anyone going to anything like this or casting as wide a net in our discussions with the public.

Of all the fields Crispr will touch, medicine is the one most primed for disruption. So its of great concern to conference-goers that Crispr doesnt become a technology only for the haves and not the have-nots. Shakir Cannon, founder of the Minority Coalition for Precision Medicine, pointed out the myriad ways doctors and researchers have exploited people of color in the name of scientific advancement, while neglecting diseases that hit underserved communities the hardest. In a breakout session on Wednesday, Rachel HaurwitzCEO of Caribou Biosciences, one of the big three Crispr companiesasked Cannon and his colleague, Michael Friend, how industry leaders could help make sure that doesnt happen. First, you have to build trust with communities, said Friend, whose work focuses on sickle cell anemia. But we think Crispr could be a real turning point.

Still, CrisprCon was just more talkwhich the field has seen a lot of recently. Crisprs co-discoverer Jennifer Doudna has taken a step back this past year from her lab at Berkeley to travel the world and discuss the importance of coming to what she calls a global consensus on appropriate uses for gene editing technologies. And in her opening address on Wednesday, the standing-room-only auditorium heard a line shes trotted out many times before. I've never seen science move at the pace its moving right now, Doudna said. Which means we cant put off these conversations." The conversations happening at CrisprCon were all the right ones. But action, whether in the form of regulations, laws, or other populist social contracts, still feels a long way off.

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Crispr Fans Dream of a Populist Future for Gene Editing | WIRED - WIRED

In BriefCRISPR co-discoverer Jennifer Doudna stressed the importance of using the technology with proper consideration at CrisprCon this week.

The CRISPR gene editing tool has already been used to perform some incredible feats of science, from manipulating the social behavior of ants to making superbugs kill themselves. Its an incredibly powerful asset, but this week at CrisprCon, there was plenty of discussion about where we should draw a line on its usage.

Ive never seen science move at the pace its moving right now, said CRISP co-discoverer Jennifer Doudna, who has spent recent months touring the world campaigning for a global consensus on appropriate implementations of gene-editing technologies. Which means we cant put off these conversations.

CRISPR has already been used to edit harmful conditions out of animals and even viable human embryos. From this point, it wouldnt take a great leap to start using the technology to enhance healthy organisms which is why now is the time for discussions about the consequences.

While medical uses of CRISPR are perhaps the most ethically urgent, the conversation about its usage goes beyond medicine. Companies like Monsanto and Cargill have already licensed CRISPR technologies to help with their agricultural efforts. However, early attempts at genetically modified crops struggled to gain mainstream acceptance, and thats something these firms need to keep in mind as they implement the latest techniques.

It was a convenience item for farmers, observed organic farmer Tom Wiley at the convention, according to Wired. And a profit center for corporations. To combat genetically modified foods perception problem, companies using CRISPR will have to make sure that the technology benefits the consumer, not just the production process.

The convention addressed CRISPR usage in many different fields: from the importance of ensuringit is used to address the widest range of medical conditions as possible, to the potentially damaging effects of gene drives on a delicate ecosystem.

Science is moving at a rapid pace, and CRISPR is too but if we dont carefully consider which applications are safe and valid, it could quickly cause as many problems as it solves.

Crispr is not a light on the nation, its a mirror, said CrisprCon keynote speaker Greg Simon, director of the Biden Cancer Initiative;Wiredreporter Megan Molteni interpreted those words as,its just another technology thats only as good as the people using it.

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CRISPR Co-Discoverer: "I've Never Seen Science Move at the Pace ... - Futurism

Science and ethics experts took part in a first-of-its-kind conference on the role of gene editing, and nearly half of the sold-out crowd was involved in the food and agriculture sector. CRISPRcon a summit named for the genome editing technique known as clustered regularly interspaced short palindromic repeats (CRISPR) brought together a diverse set of panelists to discuss this emerging technology.

CRISPR technology allows precise changes to be made to the DNA of living cells, which holds the potential to eradicate diseases, transform agriculture and enable massive leaps forward in environmental and life sciences. Through a series of keynote speakers, panels and interactive discussions, CRISPRcon offered a single forum for those with a stake in gene editing to share ideas, ask and answer questions and explore the path forward.

Since the CRISPR-Cas9 technology was invented five years ago by a team led by Dr. Jennifer Doudna, professor of chemistry and of molecular and cell biology at the University of California-Berkeley, and her colleague Emmanuel Charpentier, it has revolutionized biomedical and agricultural research while fueling angst about questionable applications, such as designer crops, farm animals and humans.

Its really a very cross-cutting technology, Doudna told attendees.

In fact, she said unlike earlier ways of manipulating genetic information in cells, the thing that makes CRISPR particularly powerful is the fact that it really is a democratizing tool. Its a technology that is easy enough to use and to employ that its accessible to a wide range of people, Doudna said.

It has been possible to globally adopt the technology for use in any organism, she added.

Doudna discussed applications of gene editing, including producing cattle with no horns, finding ways to treat human genetic diseases of the blood, cancer-related research, generating animals that would be better organ donors for humans, as well as plant and crop research.

The agriculture industry was represented among speakers. Thomas Titus, a pork producer from Illinois, was one of only two farmers who presented among the scientific experts, physicians, patients, environmentalists, consumers and community leaders.

Gene editing will have great impact on the future of farming, and especially on livestock production, Titus said. Although in its very early stages of development and acceptance, gene editing could ultimately be used to make pigs resistant to diseases, thereby improving food safety, animal welfare and the environmental impact of agriculture.

Titus, who raises pigs and also grows grain on his Illinois farm, was part of a panel discussing where CRISPR technology could take society by 2050. His appearance was supported by the pork checkoff and the National Pork Producers Council. Other panelists included representatives from the Center for Genetics & Society, the Institute for the Future, PICO National Network and The Breakthrough Institute.

Todays consumer is educated and asking questions about where their food comes from and how it is raised, Titus said. Thats why I welcome every chance I get to talk about todays pork production. I appreciated the opportunity to once again open my barn doors to share how I raise pigs with these key influencers in food production.

Other topics addressed during the conference included societal perception and acceptance of CRISPR application in surgery, human health and food production and conservation.

Doudna said just understanding the science is a challenge for many people, but then they also have to understand how the technology is going to affect them.

She encouraged scientist to take a very active role in engaging in conversation about gene editing, adding that its always challenging to explain technical work in a non-technical setting.

Its important to appreciate what the technology can and cannot do. Its not a magical technology; its not perfect, she said. While there are still a number of aspects of the technology that are still at the beginning phase, Doudna said the field is an incredibly fast-moving area. Ive never seen science move at the pace it is moving right now, she added.

When asked how to know when to use the cutting-edge technique, Doudna said the recommendation is to look for situations where there really is no other reasonable way to deal with a genetic disease other than gene editing. When you think about it that way, those situations are rare, she noted.

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Genome editing CRISPR technique takes center stage | Feedstuffs - Feedstuffs

News that U.S. scientists led by Oregon Health and Sciences University biologist Shoukrat Mitalipov have used the gene-editing technique known as CRISPR to modify the DNA of human embryos has led to renewed debate over human genetic engineering. Although scientists in China and the United Kingdom have already used gene editing on human embryos, the announcement that the research is now being done in the United States makes a U.S. policy response all the more urgent.

The scientists created 131 embryos that carried a genetic mutation that causes hypertrophic cardiomyopathya condition that can lead to sudden and unexpected heart attacks but has few other symptomsand attempted to correct the mutation in 112 of them (leaving 19 as unmodified controls).By injecting the CRISPR complex together with the sperm cells that carried the mutation, rather than injecting CRISPR into already fertilized embryos, the scientists were able to successfully correct the mutated genes in 72 percent of the embryos.Whether the embryos were successfully or unsuccessfully treated, all were destroyed after the researchers were finished with the study.

Much of the debate over CRISPR has been framed around concerns over the creation of so-called designer babieschildren genetically engineered to possess desirable traits that will then be passed on to subsequent generations. Some science writers and journalists have tried to downplay these concerns by noting that the gene editing was done only for basic research, rather than as an attempt to create a genetically engineered human. Writing in The New York Times, Pam Belluck argued that even if scientists do modify the genes of human embryos, fears that embryo modification could allow parents to custom order a baby with Lin-Manuel Mirandas imagination or Usain Bolts speed are closer to science fiction than science.

Those downplaying concerns also argue that preexisting practices such as the abortion of fetuses diagnosed with Down syndrome or the selective discarding of embryos diagnosed with genetic disease through preimplantation genetic diagnosis (PGD) are exactly the reason gene-editing

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CRISPR and the Ethics of Human Embryo Research - Foreign Affairs

ST. LOUIS Monsanto Company has forged an agreement with ToolGen, Inc., a biotechnology company specializing in genome editing, to use ToolGens CRISPR technology platform to develop agricultural products.

The companies announced Aug. 16 that they have reached a global licensing agreement for Monsanto to access ToolGens suite of CRISPR intellectual property for use in plants.

CRISPR stands for clustered regularly interspaced short palindromic repeats. Its a relatively new way to modify an organisms genome by precisely delivering a DNA-cutting enzyme to a targeted region of DNA. The resulting modification can delete or replace specific DNA pieces, thereby promoting or disabling certain traits.

The companies noted that gene-editing technologies, like CRISPR, offer agriculture researchers significant advantages over existing plant breeding and biotechnology methods due to their versatility and efficiency.

This agreement further expands Monsantos broad portfolio of gene-editing tools that can be used to develop improved and sustainable crops, said Tom Adams, Ph.D., vice president of biotechnology for Monsanto.

As a company we remain committed to the development of safe, sustainable and high-quality crops, and look forward to leveraging the CRISPR platform.

Additional terms of the agreement were not disclosed.

In January, Monsanto announced an agreement with the Broad Institute of MIT and Harvard for the nonexclusive use of its CRISPR-Cpf1 genome-editing technology, which is different from the CRISPR-Cas9 system.

Related articles:

CRISPR mushroom created at Penn State a GMO game-changerMonsanto agreement with Broad for CRISPR systemDuPont Pioneer scientists demonstrating potential of CRISPR-Cas for agricultureResearch finds probiotics may combat disease

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Monsanto adds another CRISPR platform to genetic toolbox - Farm ... - Farm and Dairy

A staple of bad science fiction, mutant ants have been more of a figment of imagination rather than scientific reality. Weve genetically altered mice and fruit flies, but growing mutant ants has eluded scientists due to the complex life cycle of the little critters. Now two teams announced that they managed to edit out certain genes from lab ants, altering their behavior.

The team from Rockefeller University published a paper outlining how they removed orco - a gene that plays a key part in an ants odor receptors. Deleting the gene by using theCRISPR-Cas9 technique resulted in the ants losing about 90% of their olfaction. This made them unable to socialize. The ants also changed in other ways, showing affected behavior. They laid very few eggs, wandered aimlessly, and showed poor parenting.

The other team, including scientists from NYU, Vanderbilt University, University of Pennsylvania, and Arizona State University, also used CRISPR to delete the orco protein in ants to affect their communication through pheromones, causing an "aberrant social behavior and defective neural development."

You can read their paper here.

Researchers modified the ability of the ants to detect pheromones though porous hairs on their antennae. Credit: Rockefeller University.

This kind of interference with the social behavior of ants is considered a success because of the difficulty in altering the nature of insects with such a sophisticated social structure. NYU Professor Claude Desplan, who was involved in one of the studies called the modified ant they created the first mutant in any social insect.

While ant behavior does not directly extend to humans, we believe that this work promises to advance our understanding of social communication, with the potential to shape the design of future research into disorders like schizophrenia, depression or autism that interfere with it, said Desplan.

Why edit ant genes at all? Daniel Kronauer, author of the Rockefeller University study, says there are interesting biologic questions you can only study in ants.

It was well known that ant language is produced through pheromones, but now we understand a lot more about how pheromones are perceived, saysKronauer. The way ants interact is fundamentally different from how solitary organisms interact, and with these findings we know a bit more about the genetic evolution that enabled ants to create structured societies.

Check out this animation of how Kronauer and his colleagues tracked color-coded ants, while using an algorithm to analyze the resulting behavior.

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Scientists Use CRISPR Gene Editing to Create the World's First ... - Big Think

We are one step closer to having pig organ transplants, a new study shows.

Using the genetic cut-and-paste tool CRISPR, scientists have removed DNA-based viruses that usually infect pig organs, raising the chances that these animal organs could be safely transplanted into human patients one day, a process known as xenotransplantation.

Still, that doesn't mean pig organ transplants are just around the corner; scientists would still need to change other elements of pig transplants to ensure the human body doesn't reject them.

Currently, there is a dramatic shortage in the number of organ transplants available for people who need them, and many people die before they receive one. Animals such as pigs could theoretically supply an unlimited source of such organs. But immune incompatibilities and viruses that are incorporated into the pig genome, called porcine endogenous retroviruses (PERVs), have made it very likely that such pig organs would never take on their own. [11 Body Parts Grown in the Lab]

To get around those PERVs, scientists at eGenesis, a bioengineering company in Cambridge, Massachusetts, used CRISPR-Cas 9, a genetic tool that cuts the genome wherever it's targeted, to remove 62 PERVs in pig cells in culture. The team then injected these cells into pig egg cells and generated baby pigs. They then used genetic testing to show that the pigs did not contain any trace of these PERVs.

"Although we have focused in this paper on the applications to xenotransplantation, we envision, more generally, that the synergistic combination of CRISPR-Cas technology with anti-apoptosis treatment may also be used to enable large-scale genome engineering in primary cells for a broad range of applications," the researchers wrote in the study, which was published yesterday (Aug. 10) in the journal Science.

Originally published on Live Science.

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Pig-to-Human Transplants: CRISPR Gene Editing May Make This Possible - Live Science

Pig organs are the same size as human organs and function pretty much the same way, but pig to human transplantation has long been an elusive goal for researchers due to fear of activating dormant viral diseases in the pigs cells.

But theres hope on the horizon, thanks to a new study out in Science today involving CRISPRd piglets. Researchers in the study used the gene-editing technology to effectively cut out a porcine endogenous retrovirus (PERV) commonly found throughout pig bodies.

The findings represent an important breakthrough in the potential for xenotransplantation, or the use of animal organs in humans.Currently there are more than 117,000 men, women and children on the donor waiting list in the U.S., 22 of whom die each day from lack of a matching donor. The ability to use a pig heart, lungs or other body parts could shore up the shortage and save numerous lives.

This is the first time researchers have been able to demonstrate they were able to inactivate PERV and open the way for xenotransplantation without cross-species contamination.

The company behind the study, eGenesis, which was founded by Harvard genome godfather George Church and Luhan Yang, says it used a technique involving a combination of CRISPR and a method preventing primary cell death during the editing process to inactivate all 62 copies of PERV in piglet embryos. Those embryos were then implanted in sows, growing to fully formed piglets, free of PERV.

CRISPR holds enormous potential to wipe out diseases in both humans and animals, upend our food system and has many other applications we likely dont see yet. Just last week, U.S. scientists were able to demonstrate they could successfully CRISPR out a faulty heart gene mutation in human embryos. However, there is still a lot to take into account before applying the technology to fully formed human beings.

eGenesis says it will continue to monitor the piglets for any long-term effects and, according to Yang, will also further engineer the PERV-free pig strain to deliver safe and effective xenotransplantation.

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CRISPR'd pigs offer hope for the human organ transplant shortage - TechCrunch

This photograph shows Ooceraea biroi workers tagged with color dots for individual behavioral tracking. Credit: Daniel Kronauer The Rockefeller University

The gene-editing technology called CRISPR has revolutionized the way that the function of genes is studied. So far, CRISPR has been widely used to precisely modify single-celled organisms and, more importantly, specific types of cells within more complex organisms. Now, two independent teams of investigators are reporting that CRISPR has been used to manipulate ant eggsleading to germline changes that occur in every cell of the adult animals throughout the entire ant colony. The papers appear August 10 in Cell.

"These studies are proof of principle that you can do genetics in ants," says Daniel Kronauer, an assistant professor at The Rockefeller University and senior author of one of the studies. "If you're interested in studying social behaviors and their genetic basis, ants are a good system. Now, we can knock out any gene that we think will influence social behavior and see its effects."

Because they live in colonies that function like superorganisms, ants are also a valuable model for studying complex biological systems. But ant colonies have been difficult to grow and study in the lab because of the complexity of their life cycles.

The teams found a way to work around that, using two different species of ants. The Rockefeller team employed a species called clonal raider ants (Ooceraea biroi), which lacks queens in their colonies. Instead, single unfertilized eggs develop as clones, creating large numbers of ants that are genetically identical through parthogenesis. "This means that by using CRISPR to modify single eggs, we can quickly grow up colonies containing the gene mutation we want to study," Kronauer says.

The other team, a collaboration between researchers at New York University and the NYU School of Medicine, Arizona State University, the Perelman School of Medicine at the University of Pennsylvania, and Vanderbilt University. , used Indian jumping ants (Harpegnathos saltator). "We chose this species because they have a peculiar feature that makes it easy to transform workers into queens," says Claude Desplan, a Silver Professor at NYU and one of the senior authors of the second study. If the queen dies, the young worker ants will begin dueling for dominance. Eventually, one of them becomes a "pseudoqueen"also called a gamergateand is allowed to lay eggs.

"In the lab, we can inject any worker embryo to change its genetic makeup," Desplan says. "We then convert the worker to a pseudoqueen, which can lay eggs, propagate the new genes, and spawn a new colony."

Desplan, co-senior author Danny Reinberg, a Howard Hughes Medical Institute investigator at NYU Langone, and Shelley Berger, the Daniel S. Och University Professor in the departments of Cell and Developmental Biology and Biology at Penn, began studying these ants several years ago as a way to learn about epigenetics, which refers to changes in gene expression rather than changes in the genetic code itself. "The queens and the worker ants are genetically identical, essentially twin sisters, but they develop very differently," Desplan says. "That makes them a good system for studying epigenetic control of development."

The gene that both research teams knocked out with CRISPR is called orco (odorant receptor coreceptor). Ants have 350 genes for odorant receptors, a prohibitively large number to manage individually. But due to the unique biology of how the receptors worka great stroke of luck, in this casethe investigators were able to block the function of all 350 with a single knockout. "Every one of these receptors needs to team up with the Orco coreceptor in order to be effective," says Waring Trible, a student in Kronauer's lab and the first author of the Rockefeller study.Once the gene was knocked out, the ants were effectively blind to the pheromone signals they normally use to communicate. Without those chemical cues, they become asocial, wandering out of the nest and failing to hunt for food.

More surprisingly, knocking out orco also affected the brain anatomy in the adult animals of both species. In the same way that humans have specialized processing centers in the brain for things like language and facial recognition, ants have centers that are responsible for perceiving and processing olfactory cues that are expanded compared to other insects. But in these ants, the substructures of these sensory centers, called the antennal lobe glomeruli, were largely missing.

"There are many things we still don't know about why this is the case," Kronauer says. "We don't know if the neurons die back in the adults because they're not being used, or if they never develop in the first place. This is something we need to follow up on. And eventually, we'd like to learn to what extent the phenomenon in ants is similar to what's going on in mammals, where brain development does depend to a large extent on sensory input."

"Better understanding, biochemically speaking, how behavior is shaped could reveal insights into disorders in which changes in social communication are a hallmark, such as schizophrenia or depression," Berger says.

In a third related study from the University of Pennsylvania, researchers led by Roberto Bonasio altered ant behavior usingthe brain chemical corazonin. When corazonin is injected into ants transitioning to become a pseudo-queen, it suppresses expression of thebrain protein vitellogenin. This change stimulated worker-like hunting behaviors, while inhibiting pseudo-queen behaviors, such as dueling and egg deposition.

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Further, when the team analyzed proteins the ant brain makes during the transition to becoming a pseudo-queen, they found that corazonin is similar to a reproductive hormone in vertebrates. More importantly, they also discovered that release of corazonin gets turned off as workers became pseudo-queens. Corazonin is also preferentially expressed in workers and foragers from other social insect species. In addition to corazonin, several other genes were expressed in a worker-specific or queen-specific way.

"Social insects such as ants are outstanding models to study how gene regulation affects behavior," says Bonasia, an assistant professor of Cell and Developmental Biology. "This is because they live in colonies comprised of individuals with the same genomes but vastly different sets of behaviors."

Explore further: 'Princess pheromone' tells ants which larvae are destined to be queens

More information: 1. Cell, Trible et al: "orco mutagenesis causes loss of antennal lobe glomeruli and impaired social behavior in ants." http://www.cell.com/cell/fulltext/S0092-8674(17)30772-9 , DOI: 10.1016/j.cell.2017.07.001

2. Cell, Yan et al: "An engineered orco mutation produces aberrant social behavior and defective neural development in ants" http://www.cell.com/cell/fulltext/S0092-8674(17)30770-5 , DOI: 10.1016/j.cell.2017.06.051

3. Cell, Gospocic et al.: "The neuropeptide corazonin controls social behavior and caste identity in ants" http://www.cell.com/cell/fulltext/S0092-8674(17)30821-8 , DOI: 10.1016/j.cell.2017.07.014

Journal reference: Cell

Provided by: Cell Press

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Researchers use CRISPR to manipulate social behavior in ants - Phys.Org

In BriefResearchers hope to find out more about the biochemistry of disorders affecting socialization, having successfully provoked asocial behavior in ants.

Researchers have used the CRISPR technique to manipulate the social activities of ants for a study that will be published in Cell. Two independent teams knocked out the orco (odorant receptor coreceptor) gene in entire colonies of ants, which negated their ability to perceive pheromone signals they use to communicate. Without those cues, they began to exhibit asocial behaviors like leaving the safety of the nest and declining to aid efforts to hunt for food.

Ants possess a whopping 350 genes for odorant receptors, but as they all need to liaise with the orco gene to be effective, they could all be knocked out at once. The two teams chose different ants based on two distinct strategies for proliferating this edit among the colony.

One group chose a species that has no queens, instead procreating using unfertilized eggs that mature as clones, producing ants that are genetically identical. CRISPR was used to edit lone eggs, which produced an entire colony with the desired modification.

Meanwhile, the other team of researchers selected a species known for an unusual trait that sees worker ants graduate to the role of egg-laying pseudoqueen in the event that the former queen dies. The chosen worker ant had its genetic makeup modified being converted into a pseudoqueen and prompted to spawn a new colony.

The social interactions of ants are fascinating because of the way a colony acts as a single entity. And as such, these amalgamate superorganisms can potentially tell us a lot about the way we humans interface with one another.Click to View Full Infographic

The researchers observed that disabling the orco gene resulted in certain substructures from the ants central processing centers going missing. These parts of the brain are essential to their olfactory communications, and its not known exactly why they disappeared. Symptomatically, this is analogous to a number of human mental disorders.

The hope is that further research into the biochemistry of ant colony behavior could reveal more about disorders that affect social communication, like depression or schizophrenia. If we can better understand this process as it occurs in an ants brain, and then that of the invisible hand moving the colony as superorganism, we might shine a light on how similar changes that affect mammals.

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Researchers Used CRISPR to Manipulate the Social Activities of Ants - Futurism

On track to file for clinical trial application(CTA) for lead program in beta-thalassemia in 2017Rapid progress in immuno-oncology including a lead program in allogeneic CAR-T cell therapyExpanded foundational and therapeutic intellectual property positionStrong financial position to support development of pipeline and fund operations

ZUG, Switzerland and CAMBRIDGE, Mass., Aug. 10, 2017 (GLOBE NEWSWIRE) -- CRISPR Therapeutics, (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the second quarter ended June 30, 2017 and provided a business update.

CRISPR Therapeutics has had a very productive first half of the year across all aspects of our business and we remain on track to achieve the goals we set at the beginning of the year, said Dr. Rodger Novak, CEO of CRISPR Therapeutics. Our lead program in hemoglobinopathies remains on track for a CTA submission in late 2017, with clinical trials beginning in 2018. We are advancing our immuno-oncology portfolio and other in vivo applications supported by the signing of key collaborations, and we remain focused on building an organization with top notch talent.

Recent Highlights and Outlook:

Lead hemoglobinopathies program remains on track to file CTA by year-end 2017, and begin clinical trials in 2018. CRISPR Therapeutics highlighted the most recent progress of its hemoglobinopathies program during the Presidential Symposium at the 22nd European Hematology Association Annual Congress in June. The data presented provided further support for CRISPRs approach of re-creating the natural condition of hereditary persistence of fetal hemoglobin (HPFH) that is protective in sickle cell disease and in beta-thalassemia. The presentation described the ability to re-create specific HPFH gene variants in the intended target tissue, human CD34+ stem cells, and demonstrated that these gene variants increase the expression of protective fetal hemoglobin. The data presented continues to support the development of the lead product candidate for beta-thalassemia and sickle cell disease and the Company remains on track to file a clinical trial application (CTA) in Europe by year-end 2017 for beta-thalassemia.

Advancing immuno-oncology program through lead allogeneic CAR-T program and collaborations. CRISPR Therapeutics is rapidly advancing its lead immuno-oncology program through pre-IND studies and process development for manufacturing. The lead program, CTX101, is an allogeneic CAR-T cell therapy being developed for the treatment of CD19-positive malignancies. CRISPR has demonstrated the use of its proprietary gene editing technology to make targeted modifications in T cells, thereby creating an allogeneic or off-the-shelf product that is designed for a broader patient population and addresses several challenges of the current generation of autologous therapies. In June, the Company announced an agreement with MaSTherCell SA, a full-service contract development and manufacturing organization, to develop and manufacture CTX101 under cGMP conditions for use in future clinical trials. Beyond its lead program, CRISPR announced a research collaboration with Neon Therapeutics (Neon) to explore the combination of its CRISPR gene editing platform with Neons neo-antigen platform to develop novel T cell therapies.

Advancing in vivo applications through in-licensing and collaborations. In May, CRISPR Therapeutics announced the signing of an exclusive license to a family of proprietary lipid nanoparticle (LNP) technologies from the Massachusetts Institute of Technology. Utilizing this technology, the Company demonstrated high-efficiency elimination of a target protein produced in the liver. These data were presented at the Cold Spring Harbor Laboratory Genome Engineering conference in July. Additionally, CRISPR and its collaborators at the University of Florida were awarded a two-year grant from Target ALS Foundation, a non-profit organization dedicated to accelerating new treatments for ALS, to support discovery and validation of CRISPR/Cas9-based therapeutic approaches for ALS and frontotemporal dementia (FTD). In April, CRISPR announced a collaboration with StrideBio, a leading developer of adeno-associated virus (AAV) based technologies, to co-develop new vectors for the in vivo delivery of CRISPR/Cas9-based therapeutics to various organ systems.

Strengthened international intellectual property around the foundational and therapeutic CRISPR/Cas9 gene editing technology. Following recent patent grants in the United Kingdom and Europe that broadly cover its in-licensed gene editing technology, CRISPR Therapeutics announced it has received a similarly broad patent from Chinas State Intellectual Property Office (SIPO), and it has recently received additional grants or notices of allowance from Australia, New Zealand and Singapore. The claims being granted in these jurisdictions are directed to CRISPR/Cas9 single-guide gene editing methods for modifying target DNA in both non-cellular and cellular settings, including cells from vertebrate animals such as human or mammalian cells as well as composition of matter and system claims for use in any setting, including claims for the use in producing medicine for treating disease. The growing international recognition of the broad applicability of CRISPRs patent applications for use in all settings, including in human and other eukaryotic cells, continues to reinforce the Companys position as a leader in the rapidly evolving gene editing industry.

In the U.S., CRISPR announced, jointly with its licensors and other sub-licensees, that it had filed the opening brief to the U.S. Court of Appeals for the Federal Circuit (the Federal Circuit) seeking reversal of a decision by the U.S. Patent and Trademark Offices Patent Trial and Appeal Board (PTAB) in an interference proceeding relating to CRISPR/Cas9 gene editing technology. In the appeal, University of California (UC) requests reversal of the PTABs decision terminating the interference between certain CRISPR/Cas9 patent claims owned by UC and claims of the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively, Broad). In parallel with the appeal, CRISPR is pursuing other patent applications in the U.S. to pursue patents claiming the CRISPR/Cas9 technology and its use in non-cellular and cellular settings, including eukaryotic cells.

Organizational growth and senior leadership additions. CRISPR Therapeutics continues to enhance its team by attracting and hiring experts across all critical functions including research and development, manufacturing, clinical operations and other areas. In June, CRISPR appointed James R. Kasinger as General Counsel. Mr. Kasinger will oversee the companys corporate legal and governance matters. Prior to joining CRISPR, Mr. Kasinger was General Counsel and Secretary at Moderna Therapeutics. Recently, in August, CRISPR announced the appointment of Dr. Tony Ho as the new Head of Research and Development for the company. Tony brings over 20 years of experience in the industry across both research and development in multiple therapeutic areas. Most recently, Tony was SVP and Head of Oncology Integration and Innovation at AstraZeneca. Currently CRISPR Therapeutics employs 114 people across its three locations. In July, the Companys global headquarters was moved from Basel to Zug, Switzerland, as approved by shareholders at the Companys recent annual meeting.

Financial Results for Three and Six Months Ended June 30, 2017 (U.S. GAAP):

As of June 30, 2017, CRISPR Therapeutics had $272.3 million in cash as compared to $315.5 million in cash as of December 31, 2016. Based on its current operating plan, CRISPR expects its existing cash resources will be sufficient to fund operating expenses and capital expenditure requirements for at least the next two years.

Three Months Ended June 30, 2017

CRISPR Therapeutics reported a net loss of $22.3 million for the three months ended June 30, 2017 as compared to a net loss of $17.2 million for the three months ended June 30, 2016. The increase in net loss of $5.1 million resulted primarily from an increase in loss from operations of $4.7 million, an increase in the provision for income taxes of $0.3 million and an increase in other expense of $0.1 million.

Collaboration revenue for the three months ended June 30, 2017 was $3.6 million, compared to $0.8 million for the three months ended June 30, 2016. The increase of $2.8 million was primarily due to an increase in research and development service revenue under our collaboration agreements with Casebia and Vertex of $1.5 million and $1.3 million, respectively.

Research and development expenses for the three months ended June 30, 2017 were $17.1 million, compared to $8.6 million for the three months ended June 30, 2016. The increase of $8.5 million was primarily attributable to increases of $3.2 million of variable process and platform development costs, $2.1 million of facilities costs including rent and utilities at our new research facility, $2.1 million of employee-related costs and $1.1 million of employee stock based compensation costs.

General and administrative expenses were $7.8 million for the three months ended June 30, 2017, compared to $8.8 million for the three months ended June 30, 2016. The decrease of $1.0 million was primarily due to decreases of $2.0 million in costs associated with a 2016 passive foreign investment company (PFIC) tax liability and $0.9 million in employee stock based compensation costs. The decreases were offset by increases of $1.0 million in employee-related costs to support our overall growth, $0.6 million in professional and consulting expenses, and $0.3 million in facilities costs including rent and utilities at our new facility.

Six Months Ended June 30, 2017

CRISPR Therapeutics reported a net loss of $43.8 million for the six months ended June 30, 2017, compared to a net loss of $25.6 million for the six months ended June 30, 2016. The increase in net loss of $18.2 million resulted primarily from an increase of $13.8 million in loss from operations, an increase of $0.5 million in the provision for income taxes, an increase of $0.3 million in the loss from equity method investment, an increase of $0.1 million in other expense and a decrease of $11.5 million on the gain on extinguishment of the convertible loan with Vertex offset by a decrease in interest expense of $8.0 million from the convertible loan with Bayer.

Collaboration revenue for the six months ended June 30, 2017 was $6.3 million, compared to $1.3 million for the six months ended June 30, 2016. The increase of $5.0 million was primarily due to an increase in research and development service revenue under our collaboration agreements with Casebia and Vertex of $2.6 million and $2.4 million, respectively.

Research and development expenses for the six months ended June30, 2017 were $31.9 million, compared to $14.6 million for the six months ended June 30, 2016. The increase of $17.3 million was primarily attributable to increases of $5.9 million in variable process and platform development costs, $4.7 million in facilities costs including rent and utilities at our new research facility, $4.5 million in employee-related costs, and $2.2 million in employee stock based compensation costs.

General and administrative expenses were $16.4 million for the six months ended June 30, 2017, compared to $14.9 million for the six months ended June30, 2016. The increase of $1.5 million was primarily due to the increases of $2.1 million in employee-related costs to support our overall growth, $1.5 million in facilities costs including rent and utilities at our new research facility, and $0.3 million in employee stock based compensation costs. The increases were offset by a reduction of $2.4 million in our 2016 PFIC tax obligation and franchise taxes on the convertible preferred stock financings.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The company's multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Additionally, CRISPR Therapeutics has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in diseases with high unmet need. The foundational CRISPR/Cas9 patent estate for human therapeutic use was licensed from the company's scientific founder Emmanuelle Charpentier, Ph.D. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking Statement

Certain statements set forth in this press release constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the intellectual property coverage and positions of the company, its licensors and third parties, and the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. You are cautioned that forward-looking statements are inherently uncertain. Although the company believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, the forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties regarding the intellectual property protection for our technology and intellectual property belonging to third parties; uncertainties inherent in the initiation and completion of preclinical studies for the Companys product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; expectations for regulatory approvals to conduct trials or to market products; and those risks and uncertainties described under the heading Risk Factors in the companys most recent annual report on Form 10-K, and in any other subsequent filings made by the company with theU.S. Securities and Exchange Commission(SEC), which are available on the SECs website atwww.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made.

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CRISPR Therapeutics Announces Second Quarter 2017 Financial Results and Provides Business Update - GlobeNewswire (press release)

On August 2nd, scientists achieved a milestone on the path to human genetic engineering. For the first time in the United States, scientists successfully edited the genes of a human embryo. A transpacific team of researchers used CRISPR-Cas9 to correct a mutation that leads to an often devastating heart condition. Responses to this feat followed well-trodden trails. Hype over designer babies. Hope over new tools to cure and curb disease. Some spin, some substance and a good dose of science-speak. But for me, this breakthrough is not just about science or medicine or the future of humankind. Its about faith and family, love and loss. Most of all, its about the life and memory of my brother.

Jason was born with muscle-eye-brain disease. In his case, this included muscular dystrophy, cerebral palsy, severe nearsightedness, hydrocephalus and intellectual disability. He lived past his first year thanks to marvels of modern medicine. A shunt surgery to drain excess cerebrospinal fluid building up around his brain took six attempts, but the seventh succeeded. Aside from those surgeries complications and intermittent illnesses due to a less-than-robust immune system, Jason was healthy. Healthy and happy very happy. His smile could light up a room. Yet, that didnt stop people from thinking that his disability made him worse off. My family and those in our religious community prayed for Jason. Strangers regularly came up to test their fervor. Prayer circles frequently had his name on their lists. We wanted him to be healed. But I now wonder: What, precisely, were we praying for?

Jasons disabilities fundamentally shaped his experience of the world. If praying for his healing meant praying for him to be normal, we were praying for Jason to become someone else entirely. We were praying for a paradox. If I could travel back in time, Id walk up to young, devout Joel and ask: How will Jason still be Jason if God flips a switch and makes him walk and talk and think like you? The answer to that question is hard. Yes, some just prayed for his seizures to stop. Some for his continued well-being. But is that true of most? Is that what I was praying for?

The ableist conflation of disability with disease and suffering is age-old. Just peruse the history of medicine. Decades of eugenic practices. Sanctioned torture of people with intellectual disability. The mutilation of otherwise healthy bodies in the name of functional or aesthetic normality. These stories demonstrate over and over again how easily biomedical research and practice can mask atrocity with benevolence and injustice with progress. Which leads me to ask: What, precisely, are we editing for?

Although muscle-eye-brain disease does not result from a single genetic variant, researchers agree that a single gene, named POMGNT1, plays a large role. Perhaps scientists will soon find a way to correct mutations in that and related genes. Perhaps people will no longer be born with it. But that means there would never be someone like Jason. Those prayers I mentioned above? Science will have retroactively answered them. That thought brings me to tears.

I wish we could cure cancer, relieve undue pain and heal each break and bruise. But I also wish for a world with Jason and people like him in it. I want a world accessible and habitable for people full stop not just the people we design. I worry that in our haste to make people healthy, we are in fact making people we want. We, who say we pray for healing, but in fact pray for others to be like us. We, who say were for reducing disease and promoting health, but support policies and practices aimed instead at being normal. We, who are often still unable to distinguish between positive, world-creating forms of disability and negative, world-destroying forms between Deafness, short stature or certain types of neurodiversity and chronic pain, Tay-Sachs or Alzheimers. It is with great responsibility that we as a society balance along the tightrope of biomedical progress. I long for us to find that balance. Ive certainly not found it for myself. Lest I forget how often weve lost it and how easy it is to fall, I hold dearly onto the living memory of Jason. I no longer pray for paradoxes, but for parity for the promise of a world engineered not for normality, but equality.

But that world will never come if we edit it away.

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Gene Editing Might Mean My Brother Would've Never Existed - TIME

Brain boost: Several regions across the brains of mice with a CHD8 mutation are larger (pink) than in controls.

Researchers have debuted two mouse models of autism made using the gene-editing tool CRISPR. Both strains lack one functional copy of CHD8, a gene with strong ties to autism1,2.

CRISPR allows researchers to quickly and efficiently insert specific mutations into single-cell mouse embryos. Several teams have used the method to make mouse models for other conditions, including Rett syndrome, an autism-related condition. The new mice represent the first use of the method to make models expressly for autism.

CHD8, a top autism candidate, was an obvious choice for this first foray: Almost all individuals with a harmful CHD8 mutation also have autism. They also have a characteristic syndrome that includes an enlarged head, gut problems and intellectual disability.

The two new strains of mice, along with three others made with conventional techniques, recapitulate some features seen in people with a CHD8 mutation. But they differ slightly from each other in their brain and behavioral features.

These variations may be due to differences in the mices genetic background, says Alex Nord, assistant professor of neuroscience at the University of California, Davis, who made one of the new CRISPR models.

Because the genetic background of people is also widely variable, the ability to make multiple mouse models of the same syndrome inexpensively is an advantage.

The continued accumulation of these data, models and reagents is going to be enormously important for the field, says Michael Talkowski, associate professor of neurology at Harvard University, who was not involved in making the mice.

Traditional methods for disabling a gene involve breeding mice for several generations in order to generate animals that carry the mutation in each of their cells. CRISPR, by contrast, allows researchers to alter the genome directly in a single-cell mouse embryo, speeding up the process and lowering costs substantially.

Since CRISPR/CAS9 has come out, making the mouse is no longer the longest part of the process, Nord says. CRISPR has shortened the timeline for engineering a mouse model from roughly two years to about six months, researchers say.

CRISPR also makes it possible to introduce mutations in mice that are otherwise genetically identical to controls. Researchers can tweak the gene in any way they like, inserting specific mutations rather than deleting genes. (Some studies have suggested that CRISPR can introduce unintended mutations, however.)

Nord and his colleagues made mice with a mutation that shuts down one copy of CHD8. The animals forget having seen an object before and fail to associate a location or sound with a shock features suggestive of memory and learning problems. They do not have social deficits or repetitive behaviors, both of which are hallmarks of autism. The researchers presented the mice at the 2016 International Meeting for Autism Research, and published their findings 26 June in Nature Neuroscience.

The brains of the Nord mice are larger than those of controls across several regions, including the cortex, hippocampus and amygdala. The mice with the biggest differences have the most trouble with learning and memory.

The researchers measured gene expression in brain tissue from the mice during gestation, at birth and in adulthood. They found hundreds of genes expressed at lower levels than in control mice. These include many genes that influence how genetic messages are spliced, or edited, into their final protein-coding sequences. Using a statistical model, the researchers concluded that splicing is altered in the mutant mice.

The researchers also tracked the expression of 141 genes associated with autism; they found 37 of these are expressed at unusually low levels in the mutant mice.

The other set of CHD8 mice made using CRISPR come from researchers at the Massachusetts Institute of Technology. These mice are just as likely as controls to approach and interact with another mouse. But unlike controls, they do not spend extra time with a mouse theyve never met before.

This could be a sign of social deficits, but it could also indicate a problem with memory, says co-lead investigator Guoping Feng, professor of brain and cognitive sciences.

The mice show no repetitive behaviors, but they show clear signs of anxiety, and avoid open spaces. They are also better than controls at learning how to balance on a rotating rod. This feature is seen in mice missing copies of other genes linked to autism, such as PTEN and NLGN3.

Overall, the findings in the Feng mice seem to match those in the Nord mice and most other CHD8 models, says Jill Silverman, assistant professor of psychiatry and behavioral sciences at the University of California, Davis. Silverman led the behavioral analysis of the Nord mice.

It was really reassuring, we saw all the same things as [the Feng team] with regards to autism-relevant behavior, Silverman says.

The Feng mice show atypical expression of genes that regulate the light-dark cycle and protein processing, among others. This is consistent with results in other CHD8 models, including the Nord mice.

Fengs team found that levels of genes involved in WNT signaling, a signaling pathway important for development, are significantly altered in the nucleus accumbens. This region, nestled deep in the brain, plays a role in sensing reward.

The researchers measured the strength of electrical currents in the nucleus accumbens in brain slices from the mutant mice, and found that excitatory signaling in the region is enhanced compared with controls. Dampening CHD8 levels specifically in the nucleus accumbens improves the mices motor learning, but not their anxiety. The study appeared 11 April in Cell Reports.

The findings implicate a reward brain region in autism, says Silverman. People with autism may not be finding social interactions as rewarding, so its a really interesting approach in that way, she says.

Feng and his team plan to use CRISPR to make mice that lack both copies of CHD8, but only in the brain. (Missing both copies throughout the body is lethal). This approach would enhance the mutations effects and make it easier to pin down CHD8s role in the brain, Feng says. His team also intends to make mice that carry each of the CHD8 mutations seen in people with autism.

Nord and his colleagues also plan to make mice with individual mutations linked to autism. These include a mutation in a genomic region that may control CHD8s expression. Because CRISPR mice are relatively inexpensive to make, Nord says, the researchers can take the risk that the mutation, and others like it, in fact do nothing.

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Mice made with CRISPR usher in new era of autism research - Spectrum

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The CRISPR//Cas9 gene editing tool has quickly earned a reputation as a revolutionary technology, and its merits support the clout. This year has, in fact, seen so many CRISPR-related breakthroughs that its worthwhile to take a step back and take in all of the many accomplishments.

1. This week, circulating reports about the successful application of gene-editing human embryos in the US were confirmed by a research paper published in Nature. The researchers corrected one-cell embryo DNA to remove the MYBPC3 gene known to cause hypertrophic cardiomyopathy (HCM), a heart disease that affects 1 in 500 people.

2. This year, scientists successfully used gene editing to completely extract HIV from a living organism, with repeated success across three different animal models. In addition to the complete removal of the virus DNA, the team also prevented the progress of acute latent infection.

3. Semi-synthetic organisms were developed by breeding E.coli bacteria with an anomalous six-letter genetic code, instead of the normal four-base sequence. Additional gene editing was implemented to ensure that the new DNA molecules were not identified as an invasive presence.

4. The CRISPR method successfully targeted the command center of cancer called the hybrid fusion which leads to abnormal tumor growths. A cut-and-paste method allowed the creation of a cancer-annihilating gene that shrinks tumors in mice carrying human prostate and liver cancer cells.

5. Scientists also slowed the growth of cancerous cells, by targeting Tudor-SN, a key protein in cell division. Its expected that this technique could also slow the growth of fast-growing cells.

6. Gene editing techniques have also made superbugs kill themselves. By adding antibiotic resistant gene sequences into bacteriophage viruses, self-destructive mechanisms are triggered which protect bacteria.

7. Gene editing may even make mosquito-born diseases an extinct phenomenon. By hacking fertility genes, scientists have gained the ability to limit the spread of mosquitoes a success they credit to CRISPRs ability to make multiple genetic code changes simultaneously.

8. Using CRISPR, researchers have edited out Huntingtons disease from mice, pushing the symptomatic progression of the condition into reverse. Experts expect this promising technique to be applied to humans in the near future.

9. Outside of the medical field, CRISPR might also provide a more abundant and sustainable biofuel. By connecting several gene-editing tools, scientists engineered algae that produce twice the biofuel material as wild (or natural) counterparts.

Flickr: Sarah Fulcher

10. Very recently, the first-ever molecular recorder was developed a gene editing process that encodes a film directly into DNA code and with this ability, scientists embedded information into an E.coli genome.

11. Last but not least, and on the macro-scale, the U.S. Defense Advanced Research Projects Agency (DARPA) invested $65 million in a project called safe genes, designed to improve the accuracy and safety of CRISPR editing techniques. In addition to serving the public interest of avoiding accidental or intentional (cue ominous music) misuse, the seven research teams will remove engineered genes from environments to return them to baseline natural levels.

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11 Amazing Feats the Gene-Editing Tool CRISPR Just Made Possible - NBCNews.com

"Significant and Exciting"

This is a significant and exciting decision by the EPO, and we view this announcement as recognition of MilliporeSigma's important contributions to the genome-editing field, MilliporeSigma CEO Udit Batra, Ph.D., said. This patent provides protection for our CRISPR technology, which will give scientists the ability to advance treatment options for the toughest medical challenges we face today.

MilliporeSigma also predicted that it would be awarded patents for the technology in other countries as well.

The European patent to MilliporeSigma comes five months after the EPO announced an intention to grant a patent broadly covering CRISPR technology to Emmanuelle Charpentier, Ph.D., a director at the Max-Planck Institute in Berlin, together with the University of California (UC), and University of Vienna.

The patent consisted of broad claims directed to the CRISPR/Cas9 single-guide gene-editing system for uses in both noncellular and cellular settings, including in cells from vertebrate animals such as human or mammalian cellsas well as composition claims for use in any setting, including claims for use in a method of therapeutic treatment of a patient. The technology has been licensed to companies that include CRISPR Therapeuticswhose co-founders include Dr. Charpentierand ERS Genomics, both of which announced the EPO decision.

Dr. Charpentier, UC, and University of Vienna are in a legal battle royal with the Broad Institute of MIT and Harvard over who invented the gene-editing platform. Late last month, the European patent holders filed a brief with the U.S. Court of Appeals for the Federal Circuit seeking to reverse the February 15 decision by the Patent Trial and Appeal Board (PTAB). The PTAB found no interference in fact between 12 patents related to CRISPR technology that list as inventor Feng Zhang, Ph.D., of the Broad, and a patent application by Dr. Charpentier and Jennifer Doudna, Ph.D., of UC Berkeley.

The #CRISPR #patent situation in Europe just got a LOT more complicated, tweeted Jacob S. Sherkow, J.D., associate professor at the Innovation Center for Law and Technology, New York Law School, who has closely followed the CRISPR legal wrangle, on August 5.

Until now, he tweeted, the EPO granting of a patent to Dr. Charpentier, UC, and the University of Vienna didn't mean Zhang couldn't get his. Now, it's unclear.

Link:
MilliporeSigma to Be Granted European Patent for CRISPR Technology - Genetic Engineering & Biotechnology News

In BriefIn 2017, the hot new gene editing technique CRISPR has made unparalleled advancements in gene engineering. Here are 11 highlights.

The CRISPR//Cas9 gene editing tool has quickly earned a reputation as a revolutionary technology, and its merits support the clout. This year has, in fact, seen so many CRISPR-related breakthroughs that its worthwhile to take a step backand take in all of the many accomplishments.

1. This week, circulating reports about the successful application of gene-editing human embryos in the US were confirmed by a research paper published in Nature. The researchers corrected one-cell embryo DNA to remove the MYBPC3 gene known to cause hypertrophic cardiomyopathy (HCM), a heart disease that affects 1 in 500 people.

2. This year, scientists successfully used gene editing to completely extract HIV from a living organism, with repeated success across three different animal models. In addition to the complete removal of the virus DNA, the team also prevented the progress of acute latent infection.

3. Semi-synthetic organisms were developed by breeding E.coli bacteria with an anomalous six-letter genetic code, instead of the normal four-base sequence. Additional gene editing was implemented to ensure that the new DNA molecules were not identified as an invasive presence.

4. The CRISPR method successfully targeted the command center of cancer called the hybrid fusion which leads to abnormal tumor growths. A cut-and-paste method allowed the creation of a cancer-annihilating gene that shrinks tumors in mice carrying human prostate and liver cancer cells.

5. Scientists also slowed the growth of cancerous cells, by targeting Tudor-SN, a key protein in cell division. Its expected that this technique could also slow the growth of fast-growing cells.

6. Gene editing techniques have also made superbugs kill themselves. By adding antibiotic resistant gene sequences into bacteriophage viruses, self-destructive mechanisms are triggered which protect bacteria.

7. Gene editing may even make mosquito-born diseases an extinct phenomenon. By hacking fertility genes, scientists have gained the ability to limit the spread of mosquitoes a success they credit to CRISPRs ability to make multiple genetic code changes simultaneously.

8. Using CRISPR, researchers have edited out Huntingtons disease from mice, pushing the symptomatic progression of the condition into reverse. Experts expect this promising technique to be applied to humans in the near future.

9. Outside of the medical field, CRISPR might also provide a more abundant and sustainable biofuel. By connecting several gene-editing tools, scientists engineered algae that produce twice the biofuel material as wild (or natural) counterparts.

10. Very recently, the first-ever molecular recorder was developed a gene editing process that encodes a film directly into DNA code and with this ability, scientists embedded information into an E.coli genome.

11. Last but not least, and on the macro-scale, the US Defense Advanced Research Projects Agency (DARPA) invested $65 million in a project called safe genes, designed to improve the accuracy and safety of CRISPR editing techniques. In addition to serving the public interest of avoiding accidental or intentional (cue ominous music) misuse, the seven research teams will remove engineered genes from environmentsto return them to baselinenatural levels.

Original post:
11 Incredible Things CRISPR Has Helped Us Achieve in 2017 - Futurism

The Potential of CRISPR

The potential of the gene editing toolCRISPRjust seems to keep growing and growing, and the latest experimental use of the technology is creating skin grafts that trigger the release of insulin and help manage diabetes.

Researchers have successfully tested the idea with mice that gained less weight and showed a reversed resistance to insulin because of the grafts (high insulin resistance is a common precursor to type 2 diabetes).

In fact, the team from the University of Chicago says the same approach could eventually be used to treat a variety of metabolic and genetic conditions, not just diabetes its a question of using skin cells to trigger different chemical reactions in the body.

We didnt cure diabetes, but it does provide a potential long-term and safe approach of using skin epidermal stem cells to help people with diabetes and obesity better maintain their glucose levels,says one of the researchers, Xiaoyang Wu.

If youre new to theCRISPR(Clustered Regularly Interspaced Short Palindromic Repeats) phenomenon, its a new and innovative way of editing specific genes in the body, using a biological copy and paste technique: it can doeverything fromcut out HIV virus DNA to slow thegrowth of cancer cells.

For this study, researchers used CRISPR to alter the gene responsible for encoding a hormone calledglucagon-like peptide-1(GLP-1), which triggers the release of insulin and then helps remove excess glucose from the blood.

Type 2 diabetescomes about due to a lack of insulin, also known as insulin resistance.

Using CRISPR, the GLP-1 gene could be tweaked to make its effects last longer than normal. The result was developed into skin grafts that were then applied to mice.

Around 80 percent of the grafts successfully released the edited hormone into the blood, regulating blood glucose levels over four months, as well as reversing insulin resistance and weight gain related to a high-fat diet.

Significantly, its the first time the skin graft approach has worked for mice not specially designed in the lab.

This paper is exciting for us because it is the first time we show engineered skin grafts can survive long term in wild-type mice, and we expect that in the near future this approach can be used as a safe option for the treatment of human patients,says Wu.

Human treatments will take time to develop but the good news is that scientists are today able to grow skin tissue very easily in the lab using stem cells, so that wont be an issue.

If we can make it safe, and patients are happy with the procedure, then the researchers say it could be extended to treat something likehaemophilia, where the body is unable to make blood clots properly.

Any kind of disease where the body is deficient in specific molecules could potentially be targeted by this new technique. And if it works with diabetes, it could be time to say goodbye to needles and insulin injections.

Other scientists who werent directly involved in the research, including Timothy Kieffer from the University of British Columbia in Canada, seem optimistic.

I do predict that gene and cell therapies will ultimately replace repeated injections for the treatment of chronic diseases, Kieffer told Rachel Baxter atNew Scientist.

The findings have been published inCell Stem Cell.

Read the original:
CRISPR Skin Grafts Could Replace Insulin Shots For Diabetes - Futurism

Existing cancer immunotherapies harness T cells that recognize and home in on tumor-specific targets and kill the cancer cells. Immunotherapy using checkpoint inhibitors, for example, disconnects immune system restraints so that the T cells can attack the cancer cells. Other forms of immunotherapy, including cancer vaccines and adoptive T-cell therapy, increase the numbers of cytotoxic T cells that are mobilized to the tumor.

Immunotherapy can be highly effective against advanced cancers in some patients, but in other cases treatment doesnt work. To try to understand the genetic basis of these differing responses, scientists at the U.S. National Institutes of Health developed a genome-scale CRISPR/Cas9 screen that allowed them to knock out every single gene in a melanoma cell line and then systematically test each gene for its effect on T-cell responses against the melanoma. Using this "two-cell type" (2CT)-CRISPR assay, the researchers, led by Shashank Patel, Ph.D., and Nicholas Restifo, M.D., who is a senior investigator with NCI's Center for Cancer Research, identified more than 100 "essential" genes that were required in the melanoma line for T cells to effectively engage with and kill the cells. When these genes were knocked out, the tumor cells were more able to resist exposure to T cells that had been engineered specifically to recognize tumor-associated antigens.

The researchers published their studies in the August 7 issue of Nature, in a paper entitled "Identification of Essential Genes for Cancer Immunotherapy. The NIH team worked in close collaboration with Feng Zhang, Ph.D., from MIT, one of the original innovators of the CRISPR technology. Neville Sanjana, Ph.D., from the New York Genome Center and New York University was co-first author of the study.

CRISPR/Cas9 screens have previously been used to identify genes that play key roles in cancer cell proliferation, drug resistance, and metastasis, the authors point out. To identify which genes in tumors are requisite for the "effector function of T cells," the team developed the 2CT-CRISPR assay, consisting of human T cells as effectors and melanoma cells as targets, to evaluate the effects of individual gene knockouts on cancer cell susceptibility to T-cell killing. Many of the hundred or so genes identified were directly involved in cytokine release, or in antigen processing and presentation, but dozens of the genes identified were not known to be required for cytotoxic T-cell-based immunotherapy.

This indicated that the loss of genes that T cells need to kill cancer cells might be at least partially responsible for why immunotherapy fails in some patients, Dr. Restifo suggested to GEN. However, we were really surprised to find dozens of tumor genes that had major impacts on tumor cell survival, which hadnt previously been linked with the ability of T cells to kill target cancer cells. Exploring potentially new signaling pathways mediated by these genes could help us to understand how T cells interact with cancer cells to bring about cell death, and how cancers can evade the immune system.

With their list of the 100 most necessary tumor genes in hand, the researchers looked at the gene expression profiles of nearly 11,500 human tumors from The Cancer Genome Atlas (TCGA) database, across 36 tumor types, to see whether loss of these tumor genes associated with decreased cytolytic activity. The analysis identified a set of 19 genes that correlated with cytolytic activity across most of the cancer types. Ten of these were inducible by interferon- (IFN), which indicated that they might be upregulated in cancers because of increased T-cell mobilization. Loss of expression of these 19 genes within tumors could diminish or extinguish the presentation of tumor antigens (including HLA-A, HLA-F, B2M, TAP1 and TAP2); T-cell co-stimulation (ICAM1, CLECL1, LILRA1 and LILRA3); or cytokine production and signaling (JAK2 and STAT1) in the tumor microenvironment that drive infiltration and activation of T cells, and thus serve as a principal mechanism in immune evasion, the researchers write in their published paper.

The team next focused on one gene, APLNR, which codes for the apelin receptor, a G protein-coupled receptor (GPCR) that hadnt previously been associated with T-cell killing of cancer, but which is known to be mutated in a number of different tumor types. They identified seven different mutations in this gene in the genetic makeup of metastatic melanoma and lung cancer patients who had failed therapy using immune checkpoint inhibitors.

When the team introduced these same mutations into a melanoma cell line, the cancer cells were more resistant to T-cell attack. And when they injected engineered melanoma cells that lacked the APLNR gene into experimental mice, the resulting tumors were resistant to checkpoint inhibitor therapy and didnt respond as well to adoptive cell transfer as tumors with a normal APLNR gene. Dr. Patel concluded that these data demonstrate that APLNR loss reduces the effectiveness of T-cell-based cancer therapies, including immune checkpoint blockade and ACT.

More work will be needed to validate the relevance of all the genes identified by the studies, Dr. Restifo stressed to GEN. We hope that this comprehensive list of genes will act as a blueprint for further study so that we can better understand tumor resistance to cancer therapies that hinge on T-cell attack. Looking at mutations in these genes in individual patients who failed immunotherapy may enable physicians to devise the most appropriate treatments for each individual patient, according to their essential gene profiles. More importantly, we are working toward developing new approaches to cancer therapy that help more patients with cancer.

The study findings also indicate that the success of cancer immunotherapy depends on the interplay between a far greater number of genes than previously thought, Dr. Restifo commented. A deeper understanding of how T cells interact with potential target cells could also help us to develop more effective treatments for infectious and autoimmune diseases.

Original post:
CRISPR Screen Identifies Top 100 Essential Genes for Cancer ... - Genetic Engineering & Biotechnology News

The European Patent Office has signaled that it intends to grant MilliporeSigma a key CRISPR patent.

vchal/shutterstock

By Jon CohenAug. 4, 2017 , 4:16 PM

MilliporeSigma, a subsidiary of pharmaceutical giant Merck KGaA of Darmstadt, Germany, has become a new major player in the complicated European patent battles over CRISPR, the revolutionary genome-editing tool.

The European Patent Office (EPO) on 27 July signaled that it intends to grant a patent to MilliporeSigma, which operates in the United States and Canada, for the use of CRISPR to splice genetic information into eukaryotic cells. Just such a knock-in strategy made headlines this week in a controversial experiment that corrected a disease-causing gene in a human embryo. The MilliporeSigma claims explicitly state that the method does not comprise a process for modifying the germ line genetic identity of a human being.

The most high-profile patent battle over the CRISPR technology pits a group led by the University of California (UC) against the Broad Institute in Cambridge, Massachusetts, and its collaborators.In that dispute over filings at the U.S. Patent and Trademark Office, UC claims its patent covers uses of CRISPR in all types of cells, whereas the Broad says only it deserves patents for the tools use in eukaryotes, which is the key marketplace for developing novel human medicines with the technology. I find it quite fascinating that most people seem to think the patent disputes are between two groups when its far more complicated than that, says Catherine Coombes, a patent attorney with HGF Limited in York, U.K., who has handled some CRISPR-related litigation but is not now involved with what she refers to as the foundational intellectual property (IP) at the center of these disputes.

As Coombes explains, there is unlikely to be a winner takes all situation in Europe. MilliporeSigma (Sigma-Aldrich in Europe) is one of six parties that filed early CRISPR claims with EPO. In Europe its quite possible for all six of the early players to have substantially overlapping rights, Coombes says.This is a good position for MilliporeSigma to be in. Theyre going to have some great foundational IP for their business, which is going to help them massively. Aside from UC, the Broad, and MilliporeSigma, the other groups include ToolGen, Vilnius University, and Harvard College.

Jacob Sherkow, a patent specialist at the New York Law School in New York City who has followed the CRISPR case closely, says hes pretty shocked by EPOs decision. The specific claims made by MilliporeSigma, he notes, closely match what the Broads lead researcher reported in a landmark Science paper in January 2013. But MilliporeSigma filed its claims 6 days before the Broad group. Thats wild, Sherkow says. Im not sure how this gets resolved. The European patent landscape is now a sight to behold.

Link:
CRISPR patent battle in Europe takes a 'wild' twist with surprising player - Science Magazine

Earlier this week, a team of scientists, led by a researcher at Oregon Health and Science University, published a paper showing its possible to alter human embryo DNA to prevent congenital disease. The study shows that CRISPR-Cas9 is certainly powerful. But in the fanfare and controversy surrounding the news, the public may have lost sight that CRISPR is also highly versatile.

Scientists are using the technology to develop effective treatment therapies for a range of diseases, including cancer, diabetes and communicable diseases. Other researchers applygene editing to solve agricultural problems,counter bioterrorism and clean up the environment.

Since CRISPR was first identified, geneticists have been adapting it in the laboratory as a tool that could be used to alter genetic codes of all living organisms. The study, published in Nature on Wednesday has incited a debate about the ethics of using CRISPR technology to alter human genes, which draws attention to the ongoing public fear that humanity will soon have the capacity to build designer babies.

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Newsweek spoke with Jennifer Doudna, a microbiologist at the University of California, Berkeley and co-discover of the breakthrough gene-editing technique, about how quickly the technology is advancing and the progress she expects to see in the future.

What do you make of the findings in the Nature study?

In a way its not a surprising study. Theres obviously been interest in the potential application of genome editing to curing genetic disease. Ultimately, if one could do this in the germline, it would be possible to get rid of disease-causing mutations at the beginning of life.

Whats really interesting here is that the study was conducted in a way that could create a path to the clinic, and to establish a procedure for doing gene editing that would be feasible in these embryos. The researchers largely achieved that.

Whats the one thing you say to people to try to assuage their the worries that were on the path to creating designer babies?

People say it wont happen in the U.S. but what about China? I am asked this question at cocktail parties. What about Asia? What about places that have fewer restrictions, and perhaps fewer cultural feelings against germline editing? Its entirely possible that there will be use of germline editing in those jurisdictions. I encourage the scientific and clinical communities around the world to not rush CRISPR to clinical research because I think it would be a shame if a powerful technology gets a black eye in the public perception, at least in terms of using it inappropriately.

Are there other ways to use this technology in a reproductive medicine setting that dont involve editing an actual embryo?

Perhaps in the not-too-distant future it will be possible to generate gametesmeaning eggs or spermfrom somatic cells in a person. Already it is possible to do this in animals. Once this is technically feasible in humans, doctors could use CRISPR for patients with a known genetic predisposition to something or certain mutations to generate gametes that could be used in an in vitro fertilization setting. This removes the issue of embryo editing, though it doesnt remove the issue of making changes that become heritable in the human germline.

Are you surprised by how fast this research has progressed?

Its been about five years since we published our paper describing the CRISPR system and how it could be used for genome editing. I never imagined back then that I would be reading this headline in the New York Times this week.

What are you working on that shows CRISPRs broad capabilities?

Im leading the Innovative Genomics Institute, a UC San Franciscopartnership aiming to bring genome editing to important problems in human health and the environment, which is aimed at bringing people who do fundamental research like me together with clinicians and plant biologists. Weve teamed up with neurosurgeons at UC San Francisco, and were developing ways to deliver gene-editing molecules into the brain. This has nothing to do with germline editing. This is therapy for neurological disease. Im very excited about the potential to use gene editing to correct mutations that could really benefit patients in the future.

We published a paper in Nature Biotechnology earlier this year showing how we can use CRISPR for editing DNA in the brains of mice. Were focused right now on Huntingtons disease and working in a couple of different animal models toinvestigate whether the approach has a therapeutic benefit in these animals. If that looks promising then we hope to make steps toward clinical trials with our partners at UCSF.

The vast majority of scientists right now who are working with gene editingand CRISPR in particularare focused on this type of application. Researchers are not trying to make heritable changes to DNA in humans. They are trying to make changes to DNA that would impact a patient in their lifetime and have a positive effect.

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Gene Editing Could Stop Cancer, Diabetes and Bioterrorism: An Interview With CRISPR Scientist Jennifer Doudna - Newsweek

In BriefChinese researchers have successfully engineered HIV-resistance in mice using CRISPR/Cas9 to replicate a naturally occurring genetic mutation. Their method could eventually help prevent humans from contracting HIV, which currently affects more than 36.7 million people worldwide.

Of the many diseases that have plagued humanity, HIV is proving to be one of the trickiest to cure. The virus ability to remain hidden in latent reservoirs makes eliminating it particularly challenging, which is why Chinese researchers decided to test a different approach. Instead of developing a drug to fight HIV, theyre working on a way to make cells immune to the virus.

In a study published inMolecular Biology, a team led by Hu Chen of the 307 Hospital of the Chinese Peoples Liberation Army and Hongkui Deng of the Peking University Stem Cell Research Centerused CRISPR/Cas9 to induce a homozygous mutation in a gene called CCR5, which encodes receptors in immune cells.

Previous studies have shown that this mutation of CCR5 can prevent HIV from entering cells, but only a small percentage of people have it naturally. Using CRISPR/Cas9, the researchers edited human fetal liver hematopoietic stem/progenitor cells (HSPCs), which were then engrafted into mice. Their research showed that this targeted approach of editing CCR5 waseffective at making T-cells more resistant to HIV.

While this study isnt the first to use edited stem cells to develop HIV-resistance in immune cells, it is the first example of using CRISPR to edit CCR5. One of the advantages of CRISPR is its high efficiency on difficult to transfect cells, Cheng and Deng told The Scientist. Using the remarkable method, they achieved a 21 to 28 percent efficiency in editing CCR5.

This isnt surprising since CRISPR is considered the most effective and efficient gene-editing tool available. One of the most recent and remarkable demonstrations of its precision was the first-ever editing of a human embryo in the U.S.. The tooleven gives us the ability torevive extinct species(if we wanted to).

As for this CCR5 study, Kamel Khalili from Temple University told The Scientist that expectation should remain in check:[It] may not be a complete cure because the virus itself is not eliminated and may shift to using the CCR4 or another receptor to spread. However, he did add, CCR5 seems to be the one Achilles heel of HIV. There may be some other targets, but for now, its the best target.

HIV affects more than 36.7 million people worldwide, 1.8 million of whom are younger than15 years old. An approach that helps humans develop a resistance or immunity to it could be our best chance at future eradication.

Link:
Researchers Used CRISPR to Successfully Increase HIV Resistance in Animals - Futurism

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Gene editing took an important step this week when a group of U.S. researchers used CRISPR-Cas9 technology to correct a genetic error in dozens of human embryos without complications. Its a significant achievement, but amidst the hype, its worth cautioning just how much work has to be done before the technology leads to a safe and effective human therapeutic. Years of clinical testing, and likely unforeseen twists and turns, lay ahead, with many more biological mysteries to solve along the way. For a dose of reality, look no further than a separate research paper also submitted this week by one of the CRISPR fields pioneers, Feng Zhang, who warned drugmakers about potential problems that could arise in human testing.

Beyond CRISPR, weve got the latest on the ongoing healthcare soap opera, details on new drug approvals, and much more. Read on below.

THIS WEEK IN GENE EDITING

In a paper published in Nature Medicine, Feng Zhang, one of the pioneers of CRISPR-Cas9 gene editing at the Broad Institute of MIT and Harvard, urged companies to analyze the DNA of patients before giving them experimental medicines that alter their genes with the breakthrough technology.

Just two days later, Nature published the details of the paper showing a team of researchers used CRISPR to correct, in dozens of human embryos, a genetic mutation known to cause a specific type of heart disease.

In related news, Bedford, MA-based Homology Medicines, a startup trying to advance a gene editing alternative to CRISPR, raised an $83.5 million Series B round, giving it a whopping $127 million in raised cash since its inception last year.

ON CAPITOL HILL

The GOPs efforts to repeal and replace the Affordable Care Act fell short last week, but while the law lives, for now, its future remains unclear. The Trump administration again threatened to withhold critical subsidies that fund part of the ACA, rattling insurers, and tried to browbeat Congress into moving forward with a new repeal and replace effort. Yet Congress appeared to have other ideas. Some indicated it was time to move on to tax reform, while others scheduled hearings to begin working toward a bipartisan fix to the current system.

-By a 94-1 vote, the U.S. Senate passed a bill that reauthorizes the fees collected from drug and medical device developers in order to fund the FDAs regulatory review of new medical products. Before that vote, the Senate passed a right to try bill that would expand dying patients access to experimental drugs.

AND AT THE FDA

The 2010 alliance between Celgene (NASDAQ: CELG) and Agios Pharmaceuticals (NASDAQ: CELG) paid dividends this week with FDA approval of acute myeloid leukemia (AML) drug enasidenib (Idhifa). The drug is the first marketed product for Agios, and one it was able to advance from discovery to finish line far quicker than the norm.

Jazz Pharmaceuticals (NASDAQ: JAZZ) was cleared to begin selling its own AML drug, branded as Vyxeos, which the company acquired when it bought Celator Pharmaceuticals last year.

The FDA approved ibrutinib (Imbruvica) for adults with chronic graft-versus-host disease. The drug, sold by Johnson & Johnson (NYSE: JNJ) and AbbVie (NYSE: ABBV), is already on the market for a variety of blood cancers.

The agency also gave the green light to a new hepatitis C medicine, glecaprevir/pibrentasvir (Mavyret), from Enanta Pharmaceuticals (NASDAQ: ENTA) and partner AbbVie.

AstraZenecas 2015 investment in Netherlands and Redwood City, CA-based Acerta Pharma may soon pay off. The FDA will decide by early next year whether to approve Acerta lymphoma drug alacabrutinib; if it does, Acertas backers would get $1.5 billion from AstraZeneca.

Eli Lilly (NYSE: LLY) plans to seek FDA approval of migraine drug lasmitidan next year now that a second Phase 3 trial has succeeded. Lilly got the drug when it bought CoLucid Pharmaceuticals in January.

An FDA advisory committee voted to recommend approval of a hepatitis B vaccine, Heplisav-B, from Dynavax Technologies (NASDAQ: [[ticker: [[NASDAQ:DVAX[]]), and shares shot up 70 percent. But on Thursday, the Berkeley, CA, company announced that the FDA wants more information about post-marketing study plans, which will delay an FDA decision until November.

FDA advisors didnt, however, recommend approval of sirukumab (Plivensia), a rheumatoid arthritis drug developed by J&J, citing safety concerns. The agency will decide the drugs fate by Sept. 23.

New details emerged showing the internal rift between FDA staffers over the agencys controversial 2016 approval of Duchenne muscular dystrophy drug eteplirsen (Exondys 51). Heres more from Undark.

NEW STARTS, FUNDINGS & DEALS

Bristol-Myers Squibb (NYSE: BMY) paid $300 million to buy Cambridge, MA, startup IFM Therapeutics and get ahold of some cancer drugs the startup has ben developing. IFM, however, kept some assets in-house and is spinning them into a new company called IFM Therapeutics LLC.

Takeda formed a new startup in Cambridge, MA, Cardurion Pharmaceuticals, which will develop drugs for heart failure and other cardiovascular diseases.

Amplyx Pharmaceuticals of San Diego raised a $67 million Series C round of fund testing of an anti-fungal drug.

San Francisco-based Invitae (NASDAQ: NVTA) reached deals to acquire Good Start Genetics and Combimatrix (NASDAQ: CBMX), moves to expand its gene testing menu to include carrier and newborn screening.

Vertitas Genetics, a Boston company that offers whole genome sequencing for less than $1,000, acquired Curoverse, also based in Boston, to help bring artificial intelligence and machine learning tools to genetic analysis.

HEME HAPPENINGS

Shares of Spark Therapeutics (NASDAQ: ONCE) climbed 16 percent after it provided the first small, early look at a trial testing an experimental gene therapy for hemophilia A; the company quickly raised $350 million in a stock offering. BioMarin Pharmaceutical (NASDAQ: BMRN) is ahead of Spark with its own hemophilia A gene therapy.

Meanwhile, Lexington, MA, and Amsterdam-based UniQure (NASDAQ: QURE) reacquired European rights to its experimental hemophilia B gene therapy from partner Chiesi.

CUTBACKS

Pain drug developer PixarBio (OTC: PXRB) slashed its headcount by 17 and relocated its headquarters from Massachusetts to New Hampshire, according to an SEC filing. But in an e-mail to Endpoints, CEO Frank Reynolds insisted that the company is in Cambridge, MA, and remains on target for an FDA decision on its drug in 2019.

Ocular Therapeutix (NASDAQ: OCUL) laid off 19 percent of its staff, a cost-saving move that follows the FDAs rejection of the Bedford, MA, companys drug delivery device for the eye.

Frank Vinluan contributed to this report.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com

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Bio Roundup: CRISPR Advances, Obamacare Lives, FDA Nods & More - Xconomy

The results of the first gene-editing experiment conducted on a human embryo in the United States were published on Wednesday, and their implications cant be overstated. A research team was able to erase the gene mutation responsible for a condition causing sudden heart failure from a one-cell human embryo.

Researchers at Oregon Health & Science University, lead by Shoukhrat Mitalipov, used a tool called the Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR a DNA cutting and splicing machine that scientists can program to target a specific sequence of our DNA. Think of it as a precise carnival claw machine.

The ability to target and edit deadly gene mutations from a human embryo means diseases could be cured before a pregnancy even starts. Like Jonas Salks miracle cure for polio, perhaps embryonic gene editing could be the new vaccination.

But when the scientific journalNaturereleased the highly anticipated report, it once again raised concern about designer babies a fear that initially began after scientists successfully transplanted mitochondrial DNA, resulting in a so-called three-parent baby. According to some headlines, scientists were on the verge of making designer babies.

After all, if science can target a gene mutation for eradication, parents selecting hair and eye color and tweaking for increased intelligence cant be far off.But while the news is indeed huge, Aldous Huxleys dystopian Brave New World isnt as close as it might feel.

CRISPR, while incredible, has yet to be perfected to yield real-life results for humans. And its less likely to lead to designer-baby kits than to be used to target known genetic disorders. In fact, the editing of a human embryo isnt even the most exciting application of CRISPR released this summer.

So how will CRISPRs capabilities affect our lives? The answer lies in the political and technical impediments it faces.

How CRISPR could fight disease

Using CRISPR to experiment successfully on a human embryo is an important scientific advancement, but in the near term the technology is more likely to be used to treat or potentially eradicate genetic diseases in children and adults. One of the diseases currently in CRISPRs crosshairs is Huntingtons disease: a fatal genetic disease that destroys nerve cells in the brain of the sufferer, leading to mental and physical deterioration.

In June a report was released showing CRISPR had targeted and reversed the occurrence of Huntingtons disease in mice. While this doesnt sound as exciting as editing the genes of a human embryo, the success of this experiment is a tangible, practical bombshell.

In the world of clinical trials, testing on an animal subject is the step before clinical trials on humans. Although researchers are a few years out from being able to conduct CRISPR trials for Huntingtons disease, other advances with the technology are moving apace.

The summer 2017 edition of Genome magazine reported that a possible therapy using CRISPR technology and targeting a rare immune disorder, granulomatous disease, or CGD, could be used on humans in the next year. Researchers successfully corrected the genetic mutation causing CGD by using some of the patients own blood-forming stem cells.

Again, mice played a pivotal role: The edited cells were injected into the mice, then as programmed by CRISPR, worked their way into the bone marrow, where neutrophils (the missing defense mechanism in CGD patients) began being produced. Corrected cells programmed to produce normal neutrophils can theoretically be reintroduced into human sufferers with the same results.

But genetic variation in the disparate human population means one CRISPR code doesnt fit all. As each disease needs its specially designed tool, each person may need additional genetic testing to see if the tool even works for their unique DNA code.

Proposed federal budget cuts might stop CRISPR before it even starts

The National Institutes of Health, nested within the Centers for Disease Control and Prevention, has a tight rein on federal funding for human embryonic stem cell line research, or hES. President Bill Clinton signed a law restricting research on human embryos that resulted in embryosdestruction. George W. Bush, fueled by his evangelical base, further restricted testing to fewer hES lines.

In 2009, President Barack Obama overturned the Bush-era law and expanded the number of lines that once again could be scientifically studied and importantly, funded through the NIH. The Trump administrations record of rejecting scientific consensus, courting the religious right and slashing the federal budget doesnt bode well for accelerating hES research, let alone for CRISPR technology.

The successful editing of a human embryo is a breakthrough worthy of celebration,but with proper funding and continued research,equally stunning advancements with CRISPR could help many more people as the technology takes aim at a widening range of genetic diseases.

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CRISPR Helped 'Edit' A Human Embryo But That's Not All It Can Do - HuffPost

The potential of the gene editing tool CRISPR just seems to keep growing and growing, and the latest experimental use of the technology is creating skin grafts that trigger the release of insulin and help manage diabetes.

Researchers have successfully tested the idea with mice that gained less weight and showed a reversed resistance to insulin because of the grafts (high insulin resistance is a common precursor to type 2 diabetes).

In fact, the team from the University of Chicago says the same approach could eventually be used to treat a variety of metabolic and genetic conditions, not just diabetes it's a question of using skin cells to trigger different chemical reactions in the body.

"We didn't cure diabetes, but it does provide a potential long-term and safe approach of using skin epidermal stem cells to help people with diabetes and obesity better maintain their glucose levels," says one of the researchers, Xiaoyang Wu.

Immunofluorescence image of a skin graft. Image: University of Chicago

If you're new to the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) phenomenon, it's a new and innovative way of editing specific genes in the body, using a biological copy and paste technique: it can do everything from cut out HIV virus DNA to slow the growth of cancer cells.

For this study, researchers used CRISPR to alter the gene responsible for encoding a hormone called glucagon-like peptide-1 (GLP-1), which triggers the release of insulin and then helps remove excess glucose from the blood.

Type 2 diabetes comes about due to a lack of insulin, also known as insulin resistance.

Using CRISPR, the GLP-1 gene could be tweaked to make its effects last longer than normal. The result was developed into skin grafts that were then applied to mice.

Around 80 percent of the grafts successfully released the edited hormone into the blood, regulating blood glucose levels over four months, as well as reversing insulin resistance and weight gain related to a high-fat diet.

Significantly, it's the first time the skin graft approach has worked for mice not specially designed in the lab.

"This paper is exciting for us because it is the first time we show engineered skin grafts can survive long term in wild-type mice, and we expect that in the near future this approach can be used as a safe option for the treatment of human patients," says Wu.

Human treatments will take time to develop but the good news is that scientists are today able to grow skin tissue very easily in the lab using stem cells, so that won't be an issue.

If we can make it safe, and patients are happy with the procedure, then the researchers say it could be extended to treat something like haemophilia, where the body is unable to make blood clots properly.

Any kind of disease where the body is deficient in specific molecules could potentially be targeted by this new technique. And if it works with diabetes, it could be time to say goodbye to needles and insulin injections.

Other scientists who weren't directly involved in the research, including Timothy Kieffer from the University of British Columbia in Canada, seem optimistic.

"I do predict that gene and cell therapies will ultimately replace repeated injections for the treatment of chronic diseases," Kieffer told Rachel Baxter at New Scientist.

The findings have been published in Cell Stem Cell.

Original post:
CRISPR Skin Grafts Could Replace Insulin Shots For Diabetes - ScienceAlert

Merck KGaA has received a Notice of Intention to Grant from the European Patent Office that its CRISPR patent will cover genomic integration.

Everyone in biotech is gunning for a piece of the CRISPR pie, and German Merckwill likely become the next company to acquire IP, adding to the pile it began building in 2012. Mercks CRISPR patent, which theEuropean Patent Office (EPO) has just signaled its intent to grant, will cover the use of the technology in a genomic integration method for eukaryotic cells, according to the statement.

This comes hours after the official publication of the results from thefirst embryo editing experiment in the US, headed by Shoukhrat Mitalipov at Oregon Health & Science University. (If youve been living under a rock, it was leaked last week.) His research group successfully replaced a gene that causes heart disease with a healthy one not only efficiently but also accurately, though the edited DNA wasnt taken up by a single embryo in the experiment.

As Richard Hynes, Professor of Cancer Research at MIT, toldThe New York Times,Weve always said in the past gene editing shouldnt be done, mostly because it couldnt be done safely. Thats still true, but now it looks like its going to be done safely soon [Its] a big breakthrough.Merck KGaA may lead the charge on this side of the Atlantic.

Filed in May of this year, Mercks patent application describes what it calls proxy-CRISPR, which improves the efficiency, flexibility, and specificity of the original technique by giving access to previously inaccessible cell locations. The pharma says explicitly that the method can be used to replace a disease-associated mutation with a beneficial or functional sequence, limiting its CRISPR patent to therapies and disease models while ruling out the introduction of vanity genes.

Ahorde of companies has been rushing into the CRISPR patent space, albeit for a broad range of comparatively narrow uses. Cellectis, for instance,just secured one for its CAR-T efforts. The battle for the original CRISPR patent is ongoing, though the EPO unsurprisingly ruled in favor of Emmanuelle Charpentier earlier this year after its American counterpart sided with Feng Zhang of The Broad Institute. For everyone missing the boat, theres always licensing.

Images via nobeastsofierce, crystal light / shutterstock.com

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Designer Babies in Europe: German Merck Set to Receive CRISPR Patent - Labiotech.eu (blog)