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CRISPR Co-Discoverer: "I’ve Never Seen Science Move at the Pace … – Futurism

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 Fans Dream of a Populist Future for Gene Editing | WIRED – WIRED

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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Genome editing CRISPR technique takes center stage | Feedstuffs – Feedstuffs

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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Former Hollywood Stuntman Raising $10,000 For His Stem Cell … – DNAinfo

Len Richard (left) and actor Terrance Howard on the set of “Empire” in 2015. View Full Caption

Provided by Len Richard

CHICAGO Len Richard used to fight in movie scenes as a stuntman, but now hes fighting for his life.

Diagnosed with liver disease in 2009, hes been placed on the liver and kidney transplant list to replace his rapidly failing organs. Hes opted to have stem cell replacement therapy in place of the transplant, but his insurance wont cover it, so hes raising $10,000 through YouCaring.

The 44-year-old Englewood native said hes desperate to live a healthy life and doesnt want to risk trying the transplant. Theres the fear that the new organs wont work for long and hell constantly get sick from the anti-rejection medications hell have to take, he said.

Once I understood how stem cell works and how it cures people, it was a no-brainer, Richard said. I rather do that than have someone’s organs.

More than 5,000 liver transplants in the United States take place each year, according to the “Stem Cell Therapy for Liver Diseases,” a review article published in the Journal of Stem Cell Research and Therapy. About 20,000 people are waiting for a transplant, but only 7,000 procedures are performed each year, and up to 1,500 patients die each year waiting.

Use of stem cells to cure liver diseases has been proved beneficial in most of the conditions, according to the article. Scientific literature reveals the role of stem cells in treatment and cure of various diseases like liver cirrhosis, end stage liver failure, genetic liver disease and also the liver cancer. The stem cells possess the ability to renew and multiply by them or stem cells possess special characteristics of regenerating themselves.

Besides being hospitalized twice in eighth grade for a high fever and an enlarged liver and spleen, Richard has lived a fairly healthy life, he said. He had no major health problems in high school or college.

As an adult, he worked behind the scenes in the control room at Channel 50, before moving to Los Angeles in 1998 for a similar television job. Thats when his life changed.

He was at a gym working out and got invited to train with a group who worked as stuntmen, he said.

A stunt coordinator saw me and said that I looked like Omar and Cuba Gooding, Richard said. He hired me for ‘Baby Boy.’ Thats how I got into doing stunts.

That job led to other jobs in major films, including “Barbershop” and “Transformers.”

Everything was going well for Richard, he said, until 2008 when he tore his rotator cuff during filming for the movie “First Sunday,” starring Ice Cube and Katt Williams.

I had surgery, and the person I was seeing at the time noticed that I started losing weight and was going to the bathroom a lot, Richard said.

When he went to the doctor for a routine checkup, he learned that he was diabetic. The doctor reviewed his medical history and asked if he has ever been evaluated for a liver transplant.

In 1987, when he was 13, he mysteriously became ill, he said. He was in the hospital for a high fever and the doctors noticed that his liver and spleen were enlarged, but didnt know why. He was sent home only to return a few days later.

I spent the whole summer in the hospital, Richard said. They did exploratory abdominal surgery and took a sample of all of my organs. They sent it to the CDC and other labs and came back with nothing.

Now that hes on the transplant list, Richard is hoping that hes able to raise enough money to travel to see a doctor in Mexico who was recommended by another patient and have the alternative procedure instead.

I want to avoid the transplant and keep my organs, Richard said.

He said he misses his old life, although he did more recently work on the show “Empire.” Hes on disability now, but wants to return to work and the gym.

I was always in the gym, used to go hiking a lot, but now I work out when I feel like it, he said. I have low energy, and its kind of hard right now. I’ll ride a bike and try to do air squats, but I was doing crossfit before it became too much for me.

Nobody wants to be sick. Id like to be back in California, moving around. I just get tired of going to doctor, getting poked and having having them tell me I need a transplant. Its mentally draining, and it’s scary.

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U.S. DOD to Start New Trial with Pluristem’s PLX-R18 Cell Therapy … – Genetic Engineering & Biotechnology News

The U.S. Department of Defenses (DOD) Armed Forces Radiobiology Research Institute (AFRRI) is to undertake a pilot study in nonhuman primates (NHP) evaluating Pluristem Therapeutics PLX-R18 as a treatment for acute radiation syndrome (ARS) prior to and within the first 24 hours of radiation exposure. The AFRRI is part of the Uniformed Services University of Health Sciences (USUHS).

Pluristems PLX-R18 is an off-the-shelf placental expanded (PLX) cell therapy product generated from placenta-derived mesenchymal-like adherent stromal cells. The cells are designed to release a combination of therapeutic proteins to help treat bone marrow that has been damaged due to ARS, or as a result of cancer, cancer therapy, or immune-mediated bone marrow failure.

Last month, Pluristem reported positive data from an ongoing Phase II-equivalent NHP trial carried out by the National Institute of Allergy and Infectious Diseases (NIAID), which also investigated PLX-R18 cells as a treatment for ARS. The results confirmed that therapy increased survival rates in irradiated NHP animals.

The NIAID study is evaluating PLX-R18 administration 24 hours after radiation exposure. The prospective AFRRI study will evaluate therapy given prior to or within the first 24 hours of radiation exposure, which Pluristem points out is a timeframe that will be more relevant to the needs of the U.S. armed forces. Data from the two parallel studies will, in addition, provide a broader understanding of the potential therapeutic effects of PLX-R18 used as a countermeasure for ARS.

The new trial will also be carried out in accordance with FDAs Animal Rule pathway, which is followed when human efficacy trials are not feasible, in this case due to the ethics of exposing humans to nuclear radiation. Product approval via the Animal Rule pathway can be granted following large animal efficacy studies and human safety data.

We are pleased to see increased interest from U.S. governmental agencies in our PLX-R18 cell therapy, noted Zami Aberman, chairman and co-CEO of Pluristem. In view of the therapeutic effects of our product and the current geopolitical situation, governments can potentially shield their citizens from the dire health effects arising from exposure to nuclear radiation, saving many lives in the process, which is our ultimate goal.

Pluristems first PLX product, PLX-PAD, is designed for treating tissues damaged by ischemia, trauma, or inflammation, by secreting therapeutic proteins that trigger tissue repair, reduce inflammation, and modulate the immune system. The product is undergoing an international Phase III study for treating critical limb ischemia. The study, which will enroll 250 patients, is supported by an $8 million grant from the European Unions Horizon 2020 program. PLX-PAD is also undergoing Phase II development for treating intermittent claudication and is separately being evaluated for additional orthopedic indications, pulmonary arterial hypertension, and pre-eclampsia.

Pluristem has separately been granted FDA clearance to start a Phase I clinical trial evaluating PLX-R18 in patients with incomplete bone marrow recovery following hematopoietic cell transplantation.

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U.S. DOD to Start New Trial with Pluristem’s PLX-R18 Cell Therapy … – Genetic Engineering & Biotechnology News

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