The gene-editing tool CRISPR is everywhere these days. Scientists are using it to try and fight cancer and treat muscular dystrophy, companies are using it in agriculture, and TV executives are even writing it into shows.

And it's ubiquity isn't surprising because CRISPR is one of the biggest scientific discoveries of our time. It tremendously improves upon earlier gene-editing techniques because it's fast, cheap, and accurate. Or so we thought. A new study published last week in Nature Methods found that CRISPR might not be as precise as researchers believed it to be. But not everyone agrees.

In the study, researchers were using a strain of mice that had mutations causing early onset retinal degenerationa disorder that blinded the mice. Using CRISPR, the researchers were able to snip out and correct a mutation in a particular gene to restore the mice's vision.

But the scientists were curious about what are known as "off-target" effects, or secondary mutations caused by CRISPR that occur away from the intended genetic target. So, they compared the DNA from two CRISPR-treated mice to that of a mouse which hadn't received the gene editing. They found a surprisingly large number of differences. Namely, the CRISPR-treated mice had around 1400 small off-target mutations and over 100 more considerable genetic alterations.

"When you read about CRISPR, the focus is on the amazing ability of this system to go in and fix a single nucleotide out of the three billion in a genome. People have been looking at off-targeting, but generally you get the sense that it's not that bad, or it's not consequential. So this definitely came as a surprise," Vinit Mahajan, a Stanford University ophthalmology professor and researcher on the project, told Seeker.

According to the paper, the researchers didn't observe any physical effects of the off-target mutations at the time. But they warned that such effects could show up later in the CRISPR-treated mice or even in their offspring.

Off-target mutations are definitely something researchers need to pay attention to. Particularly those involved in clinical studies and the ongoing human trials. But not everyone thinks the study's findings are as valid as the authors present.

"CRISPR isn't ready to be used in human gene editing."

Cara Moravec is a postdoctoral researcher at the University of Wisconsin - Madison and she uses CRISPR in her research all the time. She found a few anomalies in the study that raised some concerns for her in regards to the interpretations of the findings. She says off-target effects are a known issue with CRISPR but that this study isn't the best representation of those problems.

"I think this paper does bring to light that CRISPR isn't ready to be used in human gene editing," Moravec told Motherboard, "And there are concerns about off-target effects, but in this study they're overestimating those off-target effects because of some of the choices they made in their methods."

Moravec points to a comment left on Pubmed's listing of the article, which claims that a number of the off-targets listed in the paper are actually on-target, meaning the mutations happened on the gene the researchers were shooting for, not elsewhere. And other concerns, many shared by Moravec, are raised on UC Davis professor Paul Knoepfler's blog.

While this study brings some CRISPR limitations to the forefront, all of its claims may not stand up under scrutiny. We're definitely going to need more research to really figure out the extent of off-target mutations, when they happen, and why.

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There has been speculation over whether a patent pool for CRISPR licensing might be created, making it easier for commercial parties to get involved with the technology, according to Larry Coury, senior director of dispute resolution at Regeneron.

Courys comments were made at the Life Sciences Patent Network North America, a conference hosted by Life Sciences IP Review in Boston on Thursday, June 1.

The longer you wait [to get involved in CRISPR], the more certainty you will have. But at the same time, you will also be further behind compared to the others who did some evaluation and took a risk, said Coury, when offering his advice to commercial parties.

He added: It will depend on how important this technology is to your company, and youll have to make a decision about the most appropriate time to get in. But no matter when you decide to get in, you will have to sit down and do some negotiation.

Coury explained in the panel discussion that from a commercial point of view, he thinks it is the life sciences industrys belief that firstly, no party is going to get an exclusive licence, and secondly, every party will have the opportunity to obtain a licence.

He added: I dont think there will be lawsuits with people seeking injunctions trying to prevent other parties from using CRISPR.

As was noted in the discussion, the IP landscape for CRISPR technology remains unclear and can create confusion for companies that are looking to commercialise this technology.

The moderator of the panel, Frits Gerritzen, partner at Allen & Overy, said that the CRISPR landscape was unclear.

Jared Cohen, senior director at Alexion Pharmaceuticals, said that companies that are looking to license the technology look for certainty.

He added: There is anything but certainty right now. All you can do is look at the merits of each portfolio and come up with different conclusions.

Cohen said that, depending on how important the CRISPR technology is to a company, there are other technologies which can be considered as well.

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June 6, 2017 ISkin (three-dimensional cultured skin) derived from human iPSCs. Immunohistochemical analysis with antibodies to KERATIN 14 (KRT14), p63, cytokeratins (Pan-CK), involucrin, laminin 5, loricrin, KRT10, and filaggrin. The multilayered epidermis expressed KRT14, involucrin, laminin 5, Pan-CK, loricrin, KRT10, and filaggrin in iSkin, indicating that iPSC-keratinocytes terminally differentiate in the skin equivalents. Scale bar is 100 m. Credit: Kazuhiro Kajiwara.

Myelomeningocele is a severe congenital defect in which the backbone and spinal canal do not close before birth, putting those affected at risk of lifelong neurological problems. In a preclinical study published June 6th in Stem Cell Reports, researchers developed a stem cell-based therapy for generating skin grafts to cover myelomeningocele defects before birth. They first generated artificial skin from human induced pluripotent stem cells (iPSCs), and then successfully transplanted the skin grafts into rat fetuses with myelomeningocele.

"We provide preclinical proof of concept for a fetal therapy that could improve outcomes and prevent lifelong complications associated with myelomeningoceleone of the most severe birth defects," says senior study author Akihiro Umezawa of Japan's National Research Institute for Child Health and Development. "Since our fetal cell treatment is minimally invasive, it has the potential to become a much-needed novel treatment for myelomeningocele."

Myelomeningocele, which is the most serious and common form of spina bifida, is a neural tube defect in which the bones of the spine do not completely form. As a result, parts of the spinal cord and nerves come through the open part of the spine. A baby born with this disorder typically has an open area or a fluid-filled sac on the mid to lower back. Most children with this condition are at risk of brain damage because too much fluid builds up in their brains. They also often experience symptoms such as loss of bladder or bowel control, loss of feeling in the legs or feet, and paralysis of the legs.

Babies born with myelomeningocele usually undergo surgery to repair the defect within the first few days of life. Some highly specialized centers also offer intrauterine surgery to close the defect before the baby is born. Although prenatal surgery can improve later neurological outcomes compared with postnatal surgery, it is also associated with higher rates of preterm birth and other serious complications, underscoring the need for safe and effective fetal therapies.

To address this problem, Umezawa and his team set out to develop a minimally invasive approach for generating and transplanting skin grafts that could cover large myelomeningocele defects earlier during pregnancy, potentially improving long-term outcomes while reducing surgical risks. In particular, they were interested in using iPSC technology, which involves genetically reprogramming patients' cells to an embryonic stem cell-like state and then converting these immature cells into specialized cell types found in different parts of the body. This approach avoids ethical concerns while offering the advantages of a potentially unlimited source of various cell types for transplantation, as well as minimal risk of graft rejection by the immune system.

In the new study, the researchers first generated human iPSCs from fetal cells taken from amniotic fluid from two pregnancies with severe fetal disease (Down syndrome and twin-twin3 transfusion syndrome). They then used a chemical cocktail in a novel protocol to turn the iPSCs into skin cells and treated these cells with additional compounds such as epidermal growth factor to promote their growth into multi-layered skin. In total, it took approximately 14 weeks from amniotic fluid preparation to 3D skin generation, which would allow for transplantation to be performed in humans during the therapeutic window of 28-29 weeks of gestation.

Next, the researchers transplanted the 3D skin grafts into 20 rat fetuses through a small incision in the uterine wall. The artificial skin partially covered the myelomeningocele defects in eight of the newborn rats and completely covered the defects in four of the newborn rats, protecting the spinal cord from direct exposure to harmful chemicals in the external environment. Moreover, the engrafted 3D skin regenerated with the growth of the fetus and accelerated skin coverage throughout the pregnancy period. Notably, the transplanted skin cells did not lead to tumor formation, but the treatment significantly decreased birth weight and body length.

"We are encouraged by our results and believe that our fetal stem cell therapy has great potential to become a novel treatment for myelomeningocele," Umezawa says. "However, additional studies in larger animals are needed to demonstrate that our fetal stem cell therapy safely promotes long-term skin regeneration and neurological improvement."

Explore further: Prenatal stem cell treatment improves mobility issues caused by spina bifida

More information: Stem Cell Reports, Kajiwara et al.: "Fetal therapy model of myelomeningocele with three-dimensional skin using amniotic fluid cell-derived induced pluripotent stem cells" http://www.cell.com/stem-cell-reports/fulltext/S2213-6711(17)30220-5 , DOI: 10.1016/j.stemcr.2017.05.013

Journal reference: Stem Cell Reports

Provided by: Cell Press

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Some bodily activities, sleeping, for instance, mostly occur once every 24 hours; they follow a circadian rhythm. Other bodily functions, such as body temperature, cognitive performance and blood pressure, present an additional ...

Myelomeningocele is a severe congenital defect in which the backbone and spinal canal do not close before birth, putting those affected at risk of lifelong neurological problems. In a preclinical study published June 6th ...

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The Scientist

Was a Drop in CRISPR Firms' Stock Warranted? The Scientist ISTOCK, VCHALLast week, a study made headlines worldwide with its claim that the CRISPR-Cas9 genome-editing technique is more error prone than expected. In response, some investors chose to sell their shares in CRISPR-based biotech firms and stock ... CRISPR Therapeutics Appoints James R. Kasinger as General ...Nasdaq Insider Selling: Crispr Therapeutics AG (CRSP) CEO Sells 49384 Shares of StockSports Perspectives Could Crispr Therapeutics AG (NASDAQ:CRSP) Skyrocket? The Stock Has Too Many SellersFinance News Daily all 5 news articles »

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Jennifer Doudna was sitting in her UC Berkeley office when she got the first call from a reporter asking what she thought about scientists using Crispr to modify embryos. At the time, the embryos in question were monkeys. It was late 2014, and Doudna was just beginning to become the face of Crispr/Cas9the bacterial enzyme behind todays gene editing revolution. Since then she has fielded an ongoing avalanche of questions about the implications of her discovery. How its going to change the future of everything from medicine to agriculture to energy production. But inevitably the questions always get around to super-babies.

Today, at WIREDs 2017 Business Conference in New York, it took just a few minutes. Doudna said it was exactly this possibilityCrispr custom-designed human offspringthat made her take a step back from her own research and get involved in public discussions around the technology. For the last few years shes been speaking to scientists, politicians, and federal regulators around the world about the potential risks and rewards of Crispr. I think its really likely that in the not-too-distant future it will cure genetic disease, she said. But globally we need to come up with a consensus on moving forward in a responsible way.

In 2015, Doudna was part of a broad coalition of leading biologists who agreed to a worldwide moratorium on gene editing to the germ line, which is to say, edits that get passed along to subsequent generations. But its legally non-binding, and scientists in China have already begun experiments that involve editing the genome of human embryos. Using Crispr to cure inheritable genetic diseases is still a long way off, and fraught with ethical potholes. Which is why Doudna said people who are excited about the possibilities of Crispr shouldnt look to the clinic for its first big successes, but rather to the farm field.

When I think about where we are likely to see the biggest impacts in the shortest amount of time, I really think its going to be in agriculture, she said. Plant breeders have always been geneticists at heart. And with the precision and ease of Crispr, identifying and separating out desirable traits has the potential to speed up new crop development by several orders of magnitude. Agro-giants DuPont and Monsanto have invested in Crispr licenses to accelerate their R&D efforts toward creating crops that can withstand changing climates and new disease and pest burdens. In test plots around the world gene edited crops are already growingfrom longer-lasting potatoes and flood-resistant rice to drought-hardy corn and mildew-proof wheat, to name just a few.

As a tomato farmer, Doudna was most excited about a paper that came out just last month. In it, scientists from Cold Spring Harbor Laboratory in New York tackled some of the crops trickiest modern traits. While wild plants benefit from dropping fruitit helps seed dispersalfarmers want plants where the fruit stays on, so mechanical pickers have an easier time harvesting them. When breeders found a trait called jointless that did keep the fruit on the vine, they rushed to incorporate it into their domesticated tomato varieties. But when they crossed jointless into existing tomato breeds, the resulting plants put out all these extra branches, actually diminishing the number of fruits they produced.

Using genetics to trace back 10,000 years of tomato domestication, Cold Spring Harbor researchers discovered which genes led to that weird branching. Then they used Crispr to edit their activity. The resulttomato plants with great yields that dont drop their fruits.

For me, that really illustrates the potential for this, Doudna said. Crispr allows plant breeders to do things that would have been very difficult, sometimes impossible in the past.

The first season of Westworld wasted no time in going from hey cool, robots! to well, that was bleak. Death, destruction, android tortureits all been there from the pilot onward. Then again, on a show about a theme park staffed with sentient robotssorry, hoststhose outcomes are exactly what audiences have come to expect. If science fiction has taught folks anything, its that the machines will always rise up against humankind. But why does sci-fi always veer dystopian? Westworlds creators have a theory.

Storytelling, according to show co-creator Jonathan Nolan, serves an evolutionary purpose, allowing us to try out different realities. With sci-fi, because its so often forward-looking, were inventing cautionary tales for ourselves, Nolan said today at WIREDs 2017 Business Conference in New York. In other words, when your creations would hurt a fly, its time to start worrying.

It's not going to be a beautiful lady or a beautiful man who is going to come and be your overlord.

So does that mean Nolan and his co-creator (and wife) Lisa Joy think Westworld is a foreseeable future? Not entirely. For Nolan, the robots on his show represent more of an allegory for human behavior than a cautionary tale. And Joy sees Westworld, and sci-fi in general, as an opportunity to talk about what humanity could or should do if things start to go wrong, especially now that advancements in artificial intelligence technologies are making things like androids seem far more plausible than before. Were leaping into the age of the unfathomable, the time when machines [can do things we cant], Joy said.

Even though their show loosely taps into the dystopian trope of the AI uprising, Westworlds co-creators dont think the actual AI being developed right now will lead to total apocalypse. Instead, Joy said, little bits of AIlike Amazon predicting your purchasing needs or cars helping you drive homewill take over peoples lives piecemeal. Right now, however, these technologies dont have a moral compass yet, Nolan said, adding that thanks to the algorithms telling people what news they might want to read and what opinions they might want to hear, were in a time of artificial ignorance.

If and when true androids do arrive, though, dont expect them to look like the passing-for-human hosts of Westworld, though. We anthropomorphized what the AI will look like, Joy says. Thats not going to happen. Its not going to be a beautiful lady or a beautiful man who is going to come and be your overlord.

Ask Norman Foster what if anything hed like to change about Apples recently constructed headquarters in Cupertino, California, and hell need a moment to think. The famed architect, whose firm spent the last eight years perfecting plans for Apples glassy Campus 2, is mostly pleased with the results. But there is one thing: The only hesitation I have is in terms of the changing patterns of transportation, Foster said today at the WIRED Business Conference.

Apples headquarters feature a massive underground garage built to hold 11,000 vehicles. Today, thats an amenity. But not too far in the future, its entirely possible that cars (and garages) will be far less important. Maybe the conventional garage needs to be rethought and rethought now, Foster continued. Maybe if I had a second time around Id be putting a lot of persuasive pressure to say, Make the floor-to-floor of a car park that much bigger, so if youre not going to be filling it with cars in the future you could more easily retrofit it for more habitable space.

For Foster, the future of workplace design is dependent on this kind of flexibility. It might sound counterintuitive given Steve Jobs unrelenting attention to detail. Foster recalls when Jobs first approached him in 2009 to talk about building a new campus. He had a very clear vision in terms of what the project was, Foster said on stage. Jobs wanted the building to lie low to the ground to blend into the surrounding landscape. The late Apple CEO dictated everything from door handles to materials to the ultra-tight tolerances required throughout the building. Jobs was obsessed with glass and wanted to encourage the connection between indoors and outdoors, going as far as to build a door in the campus restaurant that could completely open in 12 seconds, eliminating the barrier to the outside world.

Its easy to assume a strict adherence to vision would stifle flexibility. The reverse is actually true, Foster said. The best buildings, he contends, require a strong point of view. They must be thoughtfully designed to adapt to the ways humans and society will inevitably change, and that requires more than just building open-plan layouts.

Ultimately, the most enduring workplaces will take into account the deep-rooted desires of the people who spend time there. Theyll prioritize smart paths of circulation to help people connect with one another. Instead of sequestering employees into glass boxes, theyll encourage them to connect with nature. To be truly competitive, architects and companies have to think beyond productivity. From the very beginning, Ive protested the idea that an office headquarters, whether its mega or micro, is only about work, Foster said. Its about lifestyle.

Andy Rubin is an excellent sharer. The longtime inventor, founder of Android, and current CEO of two companiestech incubator Playground and gadget maker Essentialbelieves strongly that the best way to invent the future is to do it together. Thats why he encourages the companies he funds and advises at Playground to share technology, he said today at WIREDs 2017 Business Conference in New York. A lot of those building blocks are pretty repetitive, Rubin said. So a lot of the work this team does is repeatable. For the same reason, Rubins already sharing details of how Essential plans to organize and orchestrate the smart home, and then help others do the same.

Essential only launched publicly at the end of May, introducing two products: a phone and a smart-home hub. The phone, Rubin told the Business Conference crowd, is a necessary first step for the company. Your phone is your main screen, he said. You probably sleep with your phone two feet away from you on your nightstand. (Guilty.) The screen in your pocket is your primary point of access for all the communication, work, and time-wasting you do in a day. Rubin wanted to make a great phone, earn space in your pocket, and then start to build new tech atop that platform.

The long-term vision for Essential, Rubin said, is more closely aligned with the Essential Home, which Rubin hopes youll put in your kitchen or living room and use to control all the connected devices where you live. Just dont call it a smart speaker, or compare it to an Amazon Echo. Its beginning to explore the relationships of the mixed-mode interface, Rubin said. You have a touchscreen and an LCD combined with speech, combined with the other sensors you have in your home like cameras and doorbells and locks.

The Homes job is to take your August Smart Lock, your Nest thermostat, your Philips Hue lights, and that Samsung fridge you bought with a huge touchscreen, and make them all work together. Right now, they dont; theyre all built on different ecosystems and platforms that refuse to talk together. Everybodys building islands, Rubin said. And theyre expecting people to plug into them. When really what has to happen isand this happened with Androidthese islands need bridges.

Rubin admitted this is hard work, and a long road. But hes even starting to think about Essentials next product. Theres Home, Phoneand maybe soon, Car. Rubin specifically mentioned the potential power of a really smart dashcam anyone can put in their car. Before he gets there, Rubin has a phone and a smart-home hub to ship. And a whole lot of bridges to build.

Google is poised to begin a grand experiment in using machine learning to widen access to healthcare. If it is successful, it could see the company help protect millions of people with diabetes from an eye disease that leads to blindness.

Last year researchers at the search and ads company announced that they had trained image recognition algorithms to detect signs of diabetes-related eye disease roughly as well as human experts. The software examines photos of a patients retina to spot tiny aneurisms indicating the early stages of a condition called diabetic retinopathy, which causes blindness if untreated.

At the 2017 WIRED Business Conference in New York City today, a leader of Googles project said that work has begun on integrating the technology into a chain of eye hospitals in India. The country is one of the many places around the world where a lack of ophthalmologists means many diabetics dont get the recommended annual screening for diabetic retinopathy, said Lily Peng, a product manager with the Google Brain AI research group.

This kind of blindness is completely preventable, but because people cant get screened, half suffer vision loss before theyre detected, she said, describing the current situation in India. One of the promises of this technology is being able to make healthcare more accessible. There are more than 400 million people worldwide with diabetes, including 70 million in India.

Peng, who is an MD, was featured on WIREDs Next List of 20 tech visionaries creating the future earlier this year.

In India, Google is working with the Aravind Eye Care System, a network of eye hospitals established in the late 1970s and credited with helping reduce the incidence of blindness caused by cataracts in the country. Aravind helped Google develop its retinal screening system by contributing some of the images needed to train its image parsing algorithms. The system uses the same deep learning technique that allows Googles image search and photo storage service do things like differentiate between dogs, cats, and people.

Googles paper last year just described the accuracy of that technology when applied to retinal images, not its use in the clinic. Peng said today that Google has just finished a clinical study in Indiameaning the technology was used in real patient carewith Aravind. Work is now under way on getting the technology into routine use with patients, she said.

Peng dismissed suggestions that while this technology might be good for patients, it could mean fewer jobs for doctors. She said Googles algorithms would instead do screening work not being done today due to skills shortages while freeing physicians for more important tasks. Theres not enough expertise to go, we need to have our specialists working on treating people who are sick, said Peng.

Visa is one of the most recognized brands in the world. Its logo is synonymous with the plastic forms of payment for which the company is still best known.

The ability to pay for things with a debit card or your smartphone instead of having to carry around cash (or something to barter) is sort of miraculous. For nearly the whole of human history, and in many parts of the world still, economies have been built on the premise of physical mediums of exchange. If Visas innovation chief Jim McCarthy has his way, Visa itself may soon become invisible.

The magic of Uber and Amazon, they made payment kind of disappear, McCarthy said at the 2017 Wired Business Conference in New York today. (Visa is the conferences main corporate sponsor.)

Naturally, McCarthy wants Visa to stay at the center of the payments ecosystem, even as the consumer-side of paying for stuff becomes less visible. And to do that, he says, Visa has focused on making it easier for tech companies like Apple and Samsung to tap into Visas services.

As an example, when you use something like Apple Pay or Samsung Pay, the software actually creates a unique card number for each of your devices. So if you have an iPhone, a Samsung Gear watch, and a debit card, each one of those has a unique card number tied back to your bank account. If one of your accounts is compromised, new numbers can be created for the devices in the background without you ever having to know about it

Eventually, that that could mean the end of having to manually change your credit card number with every different service every time you get a new card.

To make all that work, the payment apps actually have to communicate with Visas servers to generate and process card numbers. Its not hard to imagine more radical scenarios, like the ability to simply walk into a store, take what you want, and leave without having to worry about the entire payment process. When that finally happens, Visa wants to be there, making all the hard parts of sending and receiving money around the world look easy.

When David Limp thinks about the future of Alexa, the AI assistant he oversees at Amazon, he imagines a world not unlike Star Treka future in which you could be anywhere, asking anything, and an ambient computer would be there to fulfill your every need.

Imagine a world in the not-so-distant future where you could have infinite computing power and infinite storage, Limp said today at WIREDs 2017 Business Conference in New York. If you take off the constrains of servers and building up infrastructure, what could you do?

Limp, who has worked at Amazon since 2010 as the senior vice president for devices, sees Alexa as a critical part of this future. Already, you can shout Hey, Alexa, and get the assistant to tell you the weather forecast, turn off the lights, hail an Uber, or thousands of other things that Amazon and developers have trained it to do. But Limp says theres still plenty more work to be done before we live in the AI-assisted future he thinks about every day, and much of that effort has to do with training machines to better understand humans.

Since Alexa made its debut in 2014, the virtual assistant has taken lease in dedicated devices like the Echo, Tap, Echo Dot, Echo Look, Echo Show, as well as dozens of other supported devices. All that interaction with humans has given Alexa plenty of voice data to parse throughdata thats helped train the assistant to understand preferences, recognize different accents, even figure out the intent of a request without specific keywords. A year ago, if youd told Alexa to order a car, it wouldnt have understood what you meant. (What, like, order one from the Amazon Marketplace?) Now, through improved machine learning, Alexa knows what you mean and will prompt you to enable an Uber or Lyft skill so that it can summon your ride.

Of course, Alexa is far from perfect. Limp says one near-future goal would be improving Alexas understanding of anaphoraso if you ask, Whos the president of the United States? and then follow up by asking, How old is he? Alexa knows youre still talking about Donald Trump. Amazon is also tinkering with Alexas short-term and long-term memory, so that the bot can recall context from yesterdays conversation as well as the thing you asked it five seconds ago.

Those changes involve a shift toward making devices that arent personal but can work for everyone. Think more like a wall clock in the kitchen, which everyone in a household can glance at to get the time, rather than a smartphone, which is designed for one person to use.

As we design the interfaces for Alexa, whether voice or graphical, its about making it ambient and so that anybody can use it, Limp said on stage. If you ask for a timer and I ask for a timer, theyre both going to work.

In a world where devices will surround people all the time, those gadgets will have to understand what humans mean, however they choose to say it. For anyone who uses Alexa, that education is already under way: Every time someone talks to their Echo, the world inches a little bit closer to that Starship Enterprise future Limp imagines.

Urs Hlzle has a big job. As senior vice president of technical infrastructure at Google, hes in charge of the hundreds of thousands of servers in data centers spread across the planet to power the companys ever widening range of services.

Hes also the person that the companys engineers turn to when all that computing power turns out not to be enough.

Today at the 2017 Wired Business Conference in New York, Hlze explained that even with its enormous resources, Google has had to find ways to economize its operations in order to meet its ambitious goals. Most recently, he said, the company was forced to start building its own artificial intelligence chips because the companys existing infrastructure just wouldnt cut it.

Around five years ago, Jeff Dean, who ran Googles artificial intelligence group, realized that his teams technique for speech recognition was getting really good. So good in fact, that he thought it was ready to move from the lab to the real world by powering Androids voice-control system.

But when Dean and Hlzle ran the numbers, they realized that if every Android user in the world used about three minutes of voice recognition time per day, Google would need twice as much computing power to handle it all. The worlds largest computing infrastructure, in other words, would have to double in size.

Even for Google that is not something you can afford, because Android is free, Android speech recognition is free, and you want to keep it free, and you cant double your infrastructure to do that, Hlzle says.

What Google decided to do instead, Hlzle said, is create a whole new type of chip specialized exclusively for machine learning. He likens traditional CPU chips to everyday carsthey have to do a lot of things relatively well to make sure you get where your going. An AI chip, on the other hand, has to do just one thing exceptionally well.

What we built was the equivalent of a drag race car, it can only do one thing, go straight as fast as it can, he says. Everything else it is really, really bad at, but this one thing it is super good at.

Googles custom chips could handle AI tasks far more efficiently than traditional chips, which meant the company could support not just voice recognition, but a broad range of other tasks as well without breaking the bank.

This pattern has repeated itself again and again during Hlzles time at Google. He says that when he started at the company in 1999 (he was somewhere between the seventh and 11th employee hired by Google, depending on how you count), Google only had around 50 servers and was straining to support the number of search queries it received each day. But even with $25 million in venture funding, the company couldnt afford to buy enough ready-made servers to meet its growing demand.

If we had done it with the machines, the servers, that people were using, professional servers, real servers, that would have blown our $25 million in an instant, he says. It really was not an option, so we were forced to look for other ways to do the same thing more cheaply.

So Hlzle and company built their own servers out of cheap parts. Each individual server was less powerful and reliable than a professional-grade machine, but together the clusters of computers they assembled was more powerful and reliable than what they could purchased otherwise. Google didnt invent the idea of using big clusters of cheap machines in lieu of more expensive hardwarethat honor might go to the nearly forgotten search engine Inktomibut it did popularize the model by proving that it could work on a massive scale.

Hlzle and his team had to do something similar years later when it found that off-the-shelf networking gear no longer met its needs. So few companies needed switches that could support the number machines Google had that no established networking company was interested in producing them. So, once again, Hlzle and his team had to build their own gearsomething that other companies, like Facebook, now do as well.

These decisions become a lot easier if all the other alternatives are non-viable, Hlzle says. Its not necessarily that were somehow bolder or more insightful, but its actually that for many of these things in our history, it was almost a forced choice, you didnt really have a viable alternative that you could buy.

But Hlzle probably isnt giving himself enough credit. Most people, after exhausting all the viable options, would conclude that their task is impossible. When Hlzle ran out of options, he created new ones.

Yasmin Green leads a team at Googles parent company with an audacious goal: solving the thorniest geopolitical problems that emerge online. Jigsaw, where she is the head of research and development, is a think tank within Alphabet tasked with fighting the unintended unsavory consequences of technological progress. Greens radical strategy for tackling the dark side of the web? Talk directly to the humans behind it.

That means listening to fake news creators, jihadis, and cyber bullies so that she and her team can understand their motivations, processes, and goals. We look at censorship, cybersecurity, cyberattacks, ISISeverything the creators of the internet did not imagine the internet would be used for, Green said today at WIREDs 2017 Business Conference in New York.

Last week, Green traveled to Macedonia to meet with peddlers of fake news, those click-hungry opportunists who had such a sway over the 2016 presidential election in the US. Her goal was to understand the business model of fake news dissemination so that she and her team can create algorithms to identify the process and disrupt it. She learned that these content farms utilize social media and online advertisingthe same tools used by legit online publishers. [The problem of fake news] starts off in a way that algorithms should be able to detect, she said. Her team is now working on a tool that could be shared across Google as well as competing platforms like Facebook and Twitter to thwart that system.

Its mostly good people making bad decisions who join violent extremist groups.

Along with fake news, Jigsaw is intensely focused on combatting online pro-terror propaganda. Last year, Green and her team travelled to Iraq to speak directly to ex-ISIS recruits. The conversations led to a tool called the Redirect Method, which uses machine learning detect extremist sympathies based on search patterns. Once detected, the Redirect Method serves these users videos that show the ugly side of ISISa counternarrative to the allure of the ideology. At the point that they are buying a ticket to join the caliphate, she said, it was too late.

Its mostly good people making bad decisions who join violent extremist groups, Green says. So the job was: lets respect that these people are not evil and they are buying into something and lets use the power of targeted advertising to reach them, the people who are sympathetic but not sold.

Since its launch last year, 300,000 people have watched videos served up by the Redirect Methoda total of more than half a million minutes, Green said.

Beyond fake news and extremism, Greens team has also created a tool to target toxic speech in comment sections on news organizations sites. They created Perspective, a machine-learning algorithm that uses context and sentiment training to detect potential online harassment and alert moderators to the problem. The beta version is being used by the likes of the New York Times. But as Green explained, its a constantly evolving tool. One potential worry is that it could be itself biased against certain words, ideas, even tones of speech. To counteract that risk, Jigsaw decided not to open up the API to allow others to set the parameters themselves, fearing that an authoritarian regime might use the tool for full-on censorship.

We have to take measures to keep these tools from being misused, she said. Just like the internet itself, which has been used in destructive ways its creators could never have imagined, Green is aware that the solutions her team creates could also be abused. That risk is always on her mind, she says. But its not a reason to stop trying.

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Crispr May Cure All Genetic DiseaseOne Day - WIRED

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Simcoe.com

Frozen in time: why an Ontario man chose cryonic suspension Simcoe.com Brian was also known to be quite the conversationalist and could tackle any subject matter, regardless of how out of the norm it was, even the concept of living forever. Phil, who became interested in cryonics as a child, recalls talking casually about ... and more »

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Without RSRT championing gene therapy for the treatment of Rett Syndrome, we would not be where we are ... Brian Kaspar, CSO of AveXis

Trumbull, CT (PRWEB) June 08, 2017

The Rett Syndrome Research Trust (RSRT) is excited to announce that AveXis, Inc., has committed to advance a gene therapy candidate for Rett Syndrome into human clinical trials. AveXis, a pioneering clinical-stage gene therapy company, has already advanced a transformative gene therapy treatment for spinal muscular atrophy Type 1, a devastating disease that strikes infants and kills in toddlerhood. Remarkably, in a Phase 1 clinical trial, infants treated with the gene therapy achieved developmental milestones, including the ability to sit unassisted, talk and walk in some cases, achievements never seen in untreated babies with the disease. It is our shared belief that gene therapy, the introduction of healthy MECP2 genes to compensate for the mutated ones, may also produce benefits for individuals with Rett Syndrome.

This milestone represents the culmination of research originating in 2010 when RSRT funded the collaboration between Dr. Gail Mandel and Dr. Brian Kaspar, the now scientific founder and Chief Scientific Officer of AveXis, to explore the potential of gene therapy for the treatment of Rett Syndrome. Encouraged by the results of that collaboration, in 2014 RSRT conceived of, recruited the scientists for and funded the Gene Therapy Consortium to definitively evaluate the feasibility of gene therapy as a treatment for Rett Syndrome.

The Consortium scientists, Stuart Cobb, Steve Gray, Brian Kaspar and Gail Mandel, exceeded our expectations by developing a gene therapy product candidate with impressive efficacy, safety and delivery characteristics. Importantly, therapeutic benefits in the mouse models are much greater than that of any drug ever tested for Rett Syndrome.

Adrian Birds 2007 reversal study showing that symptoms in mice can be dramatically reversed suggests that a diagnosis of Rett need not translate to lifelong disability. While gene therapy seems an obvious approach for any of the numerous single gene disorders, the quality of the data generated by the Consortium was an essential factor in AveXis decision to prioritize development of a gene therapy for Rett Syndrome.

RSRT recognized early on that the gene therapy approach would move forward only if we aggressively championed it. We brought together scientists with strong track records of success and provided them with the financial support and infrastructure to work effectively and efficiently. Our strategy worked and I am thrilled that AveXis, a leader in gene therapy, is now taking this work forward at an industry scale. I am beyond grateful to the many Rett families who believe in our vision and to the generosity of their networks that make our vital work possible, said Monica Coenraads, Executive Director of RSRT and mother to a young woman with Rett Syndrome.

As was made clear in RSRTs recently launched strategic research plan, Roadmap to a Cure, our mission is a cure for Rett Syndrome. Todays announcement is a huge step forward towards our goal. By addressing the core cause of Rett, gene therapy has the potential to be a life-changer. We have complete confidence in the expertise and leadership of the AveXis team and will continue to provide our full support as this program moves forward, said Randall Carpenter, Chief Scientific Officer, RSRT.

Without RSRT championing gene therapy for the treatment of Rett Syndrome, we would not be where we are preclinically in our understanding of the potential for gene therapy to treat this devastating disease, said Brian Kaspar, Chief Scientific Officer of AveXis. Bringing our expertise and focus on rare monogenic diseases, we are excited by the possibility that gene therapy may address the needs of individuals with Rett Syndrome.

About the Rett Syndrome Research Trust The Rett Syndrome Research Trust (RSRT) is a nonprofit organization with a highly personal and urgent mission: a cure for Rett Syndrome and related MECP2 disorders. In March of 2017 RSRT announced Roadmap to a Cure, a three-year, $33 million strategic research plan. The plan prioritizes four curative approaches with gene therapy as our lead program. RSRT operates at the nexus of global scientific activity enabling advances in knowledge and driving innovative research through constant engagement with academic scientists, clinicians, industry, investors and affected families. Since 2008, RSRT has awarded $42 million to research. To learn more, please visit http://www.reverserett.org.

About AveXis, Inc. AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The companys initial proprietary gene therapy candidate, AVXS-101, recently completed a Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit http://www.avexis.com.

About Rett Syndrome Rett Syndrome is a genetic neurological disorder that almost exclusively affects girls. It strikes randomly, typically at the age of 12 to 18 months, and is caused by random mutations of the MECP2 gene on the X chromosome. Rett Syndrome is devastating as it deprives young children of speech, hand use, normal movement often including the ability to walk. As the children enter childhood the disorder brings anxiety, seizures, tremors, breathing difficulties, severe gastrointestinal issues. While their bodies suffer, it is believed that their cognitive abilities remain largely intact. Although most children survive to adulthood, they require total round-the-clock care.

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AveXis to Advance Gene Therapy Program Initiated by the Rett Syndrome Research Trust - PR Web (press release)

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Posted on June 6, 2017, 6 a.m. in Allergy Gene Therapy Immune System

Using gene therapy, scientists have been able to 'turn-off' the immune response which causes allergic reaction in animals.

It might soon be possible for a single treatment to provide life-long protection against harsh allergies including asthma. An immunology research team at The University of Queensland led by Professor Ray Steptoe has figured out how to disable the immune response that triggers allergic reactions. The research team operates out of the university's Diamantina Institute. Professor Steptoe's lab is situated at the Translational Research Institute. The research was funded by the National Health and Medical Research Council and the Asthma Foundation. The research team's findings are published in JCI Insight.

The Basics of Allergies and Asthma

When an individual has an allergy or a flare-up of asthma, the symptoms he experiences stem from immune cell reactions to proteins within the allergen. Allergies and asthma recur over and over again as the immune cells, referred to as T-cells, gradually create a type of immune memory. As a result, they resist treatments. Steptoe and his research team are now capable of wiping the memories of T-cells in animals. They have successfully done so with gene therapy that desensitizes the immune system to allow for the tolerance of pain.

About the Breakthrough

Steptoe's research team made use of an experimental asthma allergen. They took blood stem cells, inserted a gene that regulates the allergen protein and put it into the recipient. These engineered cells generated new blood cells. The protein is expressed in these new blood cells. Specific immune cells are targeted in order for the allergic response to be turned off.

The experimental asthma allergen worked so effectively that it is possiblethe research could be used to treat those who suffer from traditional allergies to foods. Examples include allergies to nuts, shellfish, bee venom and an array of other substances. Professor Steptoe indicates the findings will soon be subjected to additional pre-clinical investigation. The next step is to replicate the results with human cells in a lab setting.

The Goal of Gene Therapy in the Context of Allergies

Professor Steptoe states the end goal is to make use of single injected gene therapy rather than repeated short-term treatments that attempt to reduce allergy symptoms. Such short-term treatments are successful in some instances and unsuccessful in others.

Professor Steptoe's team has not reached the point where gene therapy is as straightforward as receiving a flu jab yet his group is hard at work on making it as simple and safe as possible. Their aim is for gene therapy to be used on an extensive cross-section of those plagued by allergies and asthma as well as those who endure potentially deadly food allergies. It is possible that a completely safe one-off style gene therapy treatment for traditional allergies, asthma, and food allergies will be available in the near future.

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Allergy Breakthrough with Gene Therapy - Anti Aging News

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Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer that's on the rise. Although it's early and the study is small 35 people every patient responded and all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

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Dr. Jon LaPook introduces us to the next frontier of cancer treatment: immunotherapy, in which the body's own immune system is genetically modifi...

Experts at an American Society of Clinical Oncology conference in Chicago, where the results were announced Monday, say it's a first for multiple myeloma and rare for any cancer treatment to have such success.

Chemotherapy helps 10 to 30 percent of patients; immune system drugs, 35 to 40 percent at best, and some gene-targeting drugs, 70 to 80 percent, "but you don't get to 100," said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

"These are impressive results" but time will tell if they last, he said.

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, and more than 115,000 worldwide. It's the second fastest growing cancer for men and the third for women, rising 2 to 3 percent per year, according to the National Cancer Institute. About 60,000 to 70,000 Americans have it now.

Nine new drugs have been approved for it since 2000 but they're not cures; only about half of U.S. patients live five years after diagnosis.

With cell therapy, "I can't say we may get a cure but at least we bring hope of that possibility," said Dr. Frank Fan. He is chief scientific officer of Nanjing Legend Biotech, a Chinese company that tested the treatment with doctors at Xi'an Jiaotong University.

The treatment, called CAR-T therapy, involves filtering a patient's blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer and then given back to the patient intravenously.

Doctors call it a "living drug"- a one-time treatment to permanently alter cells that multiply in the body into an army to fight cancer. It's shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma, in patients whose cancer worsened despite many other treatments.

In the Chinese study, 19 of 35 patients are long enough past treatment to judge whether they are in complete remission, and 14 are. The other five had at least a partial remission, with their cancer greatly diminished. Some are more than a year past treatment with no sign of disease.

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Doctors in London say one-year-old Layla Richards is thriving after receiving an experimental gene therapy treatment for leukemia. CBS News' Tin...

Most patients had a group of side effects common with this treatment, including fever, low blood pressure and trouble breathing. Only two cases were severe and all were treatable and temporary, doctors said.

The second study was done in the U.S. by Bluebird Bio and Celgene, using a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients. Eighteen are long enough from treatment to judge effectiveness, and all 15 who got an adequate amount of cells had a response. Four have reached full remission so far, and some are more than a year past treatment.

The results are "very remarkable" not just for how many responded but how well, said Dr. Kenneth Anderson of Dana-Farber Cancer Institute in Boston.

"We need to be looking for how long these cells persist" and keep the cancer under control, he said.

Dr. Carl June, a University of Pennsylvania researcher who received the conference's top science award for his early work on CAR-T therapy, said "it's very rare" to see everyone respond to a treatment. His lab also had this happen - all 22 children testing a new version of CAR-T for leukemia responded, his colleagues reported at the conference.

"The first patients we treated in 2010 haven't relapsed," June said.

Dr. Michael Sabel of the University of Michigan called the treatment "revolutionary."

"This is really the epitome of personalized medicine," extending immune therapy to more types of patients, he said.

Legend Biotech plans to continue the study in up to 100 people in China and plans a study in the U.S. early next year. The treatment is expected to cost $200,000 to $300,000, and "who's going to pay for that is a big issue," Fan said.

"The manufacturing process is very expensive and you can't scale up. It's individualized. You cannot make a batch" as is done with a drug, he said.

Nick Leschly, Bluebird's chief executive, said the next phase of his company's study will test what seems the ideal dose in 20 more people.

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Editors note: The SpaceX Falcon 9 rocket scheduled to launch today from Florida was delayed due to weather conditions. The launch occured on Saturday, June 3.

A SpaceX rocket wasslated to launch two University of Colorado Boulder-built payloads to the International Space Station (ISS) from Florida on Thursday, including oneto look at changes in cardiovascular stem cells in microgravity that may someday help combat heart disease on Earth.

The Dragon spacecraft

The second payload will be used for rodent studies testing a novel treatment for bone loss in space, which has been documented in both astronauts and mice. The two payloads were developed by BioServe Space Technologies, a research center within the Ann and H.J Smead Department of Aerospace Engineering,

We have a solid relationship with SpaceX and NASA that allows us to regularly fly our flight hardware to the International Space Station, said BioServe Director Louis Stodieck. The low gravity of space provides a unique environment for biomedical experiments that cannot be reproduced on Earth, and our faculty, staff and students are very experienced in designing and building custom payloads for our academic, commercial and government partners.

The experiments will be launched on a SpaceX Falcon 9 rocket from Cape Canaveral, Florida, and carried to the ISS on the companys Dragon spacecraft. The SpaceX-CRS-11 mission launching Thursday marks BioServes 55th mission to space.

The cardiovascular cell experiments, designed by Associate Professor Mary Kearns-Jonker of the Loma Linda University School of Medicine in Loma Linda, California, will investigate how low gravity affects stem cells, including physical and molecular changes. While spaceflight is known to affect cardiac cell structure and function, the biological basis for such impacts is not clearly understood, said BioServe Associate director Stefanie Countryman.

As part of the study, the researchers will be comparing changes in heart muscle stem cells in space with similar cells simultaneously cultured on Earth, said Countryman. Researchers are hopeful the findings could help lead to stem cell therapies to repair damaged cardiac tissue. The findings also could confirm suspicions by scientists that microgravity speeds up the aging process, Countryman said.

For the heart cell experiments, BioServe is providing high-tech, cell-culture hardware known as BioCells that will be loaded into shoebox-sized habitats on ISS. The experiments will be housed in BioServes Space Automated Bioproduct Lab (SABL), a newly updated smart incubator that will reduce the time astronauts spend manipulating the experiments.

The second experiment, created by Dr. Chia Soo of the UCLA School of Medicine, will test a new drug designed to not only block loss of bone but also to rebuild it.

The mice will ride in a NASA habitat designed for spaceflight to the ISS. Once on board, some mice will undergo injections with the new drug while others will be given a placebo. At the end of the experiments half of the mice will be returned to Earth in SpaceXs Dragon spacecraft and transported to UCLA for further study, said Stodieck, a scientific co-investigator on the experiment.

BioServes Space Automated Byproduct Lab

In addition to the two science experiments, BioServe is launching its third SABL unit to the ISS. Two SABL units are currently onboard ISS supporting multiple research experiments, including three previous stem cell experiments conducted by BioServe in collaboration with Stanford University, the Mayo Clinic and the University of Minnesota.

The addition of the third SABL unit will expand BioServes capabilities in an era of high-volume science on board the ISS, said Countryman.

BioServe researchers and students have flown hardware and experiments on missions aboard NASA space shuttles, the ISS and on Russian and Japanese government cargo rockets. BioServe previously has flown payloads on commercial cargo rockets developed by both SpaceX, headquartered in Hawthorne, California, and Orbital ATK, Inc. headquartered in Dulles, Virginia.

Since it was founded by NASA in 1987, BioServe has partnered with more than 100 companies and performed dozens of NASA-sponsored investigations. Itspartners include large and small pharmaceutical and biotechnology companies, universities and NASA-funded researchers, and investigations sponsored by the Center for the Advancement of Science in Space, which manages the ISS U.S. National Laboratory. CU-Boulder students are involved in all aspects of BioServe research efforts, said Stodieck.

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SpaceX launches CU-built heart, bone health experiments to space station - CU Boulder Today

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Photonics.com Jun 2017 MINNEAPOLIS, June 6, 2017 A new 3D-laser-printed patch has been developed that can help heal scarred heart tissue after a heart attack.

Researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

"This is a significant step forward in treating the No. 1 cause of death in the U.S.," said Brenda Ogle, an associate professor of biomedical engineering at the University of Minnesota. "We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years."

The patch is modeled after a digital 3D scan of the structural proteins of native heart tissue. It is then made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells.

"We were quite surprised by how well it worked, given the complexity of the heart," Ogle said. "We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch."

The researchers will soon begin working on a larger patch and testing it on a pig heart, which is similar to a human heart.

The research study is published in the American Heart Association journal Circulation Research (doi: 10.1161/CIRCRESAHA.116.310277).

Link:
3D-Printed Patch Mends Hearts - Photonics.com

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TAMPA Researchers at the University of South Florida show in a new study that bone marrow stem cell transplants helped improve motor functions and nervous system conditions in mice with the disease amyotrophic lateral sclerosis (ALS) by repairing damage to the blood-spinal cord barrier.

In a study recently published in the journal Scientific Reports, researchers in USFs Center of Excellence for Aging and Brain Repair say the results of their experiment are an early step in pursuing stem cells for potential repair of the blood-spinal cord barrier, which has been identified as key in the development of ALS.

USF Health Professor Svitlana Garbuzova-Davis, PhD, led the project.

Using stem cells harvested from human bone marrow, researchers transplanted cells into mice modeling ALS and already showing disease symptoms. The transplanted stem cells differentiated and attached to vascular walls of many capillaries, beginning the process of blood-spinal cord barrier repair.

The stem cell treatment delayed the progression of the disease and led to improved motor function in the mice, as well as increased motor neuron cell survival, the study reported.

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Mice with ALS improve with stem cell therapy - The Ledger

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Vancouver surgeon and UBC professorRonald Lett is appealing tothe public forhelp in finding a bone marrow transplant for his wife Elizabeth Nega, who has an aggressive form of leukemia.

Nega, better known as Elsa, discovered that she had acute lymphoblasticleukemia in February and urgently needs a bone marrow transplant.However, the Ethiopian Canadian wife and mother of two has been unable to find a match because of the low number of African donors.

Ronald and Elsa are now reaching out to people of African descent to register as bone marrowdonors. They've started a website, match4elsa.com, as well as Facebook and Twitter accounts, to find Elsa and other African-Canadians life saving transplants.

"I love to live. I want to be with my kids. I want to smile again. I want to play with them again. If you save my life, you will save my whole family," said Elsa Nega in her video appeal for a donor.

Lett is the founder and international director of the charity, Canadian Network for International Surgery(CNIS). He met Elsa in Ethiopia while he was there training local doctors to perform essential surgeries.

After dedicating his life to helping others, Lett says being unable to help his wife in her time of need has been difficult.

"I helplessly watch as the love of my life suffers terribly, has devastating complications from her treatmentbut has no promise of a cure," said Lett.

"Transplant, which only works half the time, is our only hopeand all the news concerning a match for Elsahas been bad too."

Elizabeth Nega, Ronald Lett and their two children are running out of time to find Elsa a bone marrow donor. (Helen Goddard)

Since discovering that she had leukemia, Elsahas beenput through several rounds of chemotherapy, but after failing to go into remission, obtaining stem cells from a bone marrow transplant has become her only hope of recovery.

Her brother and sister in Ethiopia were her best chance, but neither were a match.

The larger issue in finding a donor for Elsa is the lack of diversity in the donor registry.

Of the 405,000 Canadians on the stem cell registry, only 800 have an African background, and none are a match for Elsa, according toChrisvanDoomwith the One Match Program.

Even among the 29 million people on the international registry, no match has been found.

Lett and Elsa's children, Lana, 8, and Lawrence, 6, have contributed to the effort.

They're in a video reading a letter appealing to Ethiopians around the world, including Canadian-Ethiopian R & B singerThe Weeknd, asking for help to save their mom.

In the meantime, Elsa's health is declining, and she's hoping for a miracle, even if it's not for her.

"If they save somebody, that's like a lotteryor a big blessing, you know.It's a big chance to get somebody to match to you and save your life.You know many people can't do this." saidNega.

People interested in registering to be a bone marrow donor can register at blood.ca,must be between 17 and 35 years old and in good health.

The test involves a cheek swab at the nearest clinicor a kit can be mailed out.

Link:
Vancouver woman's family pleading for help finding a bone marrow donor - CBC.ca

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Dr Bishesh Poudyal of the Civil Service Hospital in Kathmandu is the doctor who carried out all 18 transplants. At Civil the cost per transplant is between Rs 400,000 to Rs 500,000. KATHMANDU, June 7:A total of 18 bone marrow transplants have been successfully carried out in Nepal by a single doctor in Kathmandu since 2012.

A bone marrow transplant is a medical procedure performed to replace bone marrow that has been damaged or destroyed by disease, viral infection, or chemotherapy. This procedure involves transplanting blood stem cells, which travel to the bone marrow where they produce new blood cells and promote growth of new marrow.

Bone marrow is the spongy, fatty tissue inside the bones. It creates the red blood cells that carry oxygen and nutrients throughout the body, white blood cells that fight infection, and platelets that are responsible for the formation of clots.

Dr Bishesh Poudyal of the Civil Service Hospital in Kathmandu is the doctor who carried out all 18 transplants. "I am going to carry out bone marrow transplants on another six patients in near future," said Poudyal, who was born at Jawalakhel of Lalitpur.

Dr Poudyal, who passed SLC 24 years ago from Adarsha Vidya Mandir, was inspired by his father to pursue studies in hematology and bone marrow transplant. After completing his MBBS from China and MD from India under government scholarships, he started working at the Bir Hospital. "I served there for two years at Bir Hospital as per the government rule for scholarship students," he said.

Then, Dr Poudyal left the Bir Hospital as he came to know that bone marrow transplant was not possible at Bir and joined Civil Service Hospital. He also practised at the Nobel Medical College Hospital at Sinamangal where he started bone marrow transplant in 2012. "As I came to know Nobel was charging patients between Rs 800,000 to Rs 1 million per transplant, I quit the hospital," he said.

At his initiation, the Civil Hospital started bone marrow transplant about a year ago. At Civil the cost per transplant is between Rs 400,000 to Rs 500,000. The transplant recepients ranged from 22 years old to 64 years. Two patients died after about nine months of transplant. "One died of tuberculosis infection and another died of disease complications," according to Dr Poudyal.

"Bone marrow is transplanted in cancer and other blood diseases. Bone marrow is transplanted in different ways-- by treating patients' bone marrow, using siblings' and parents' bone marrow and matched unrelated donor (MUD). "We have not transplanted bone marrow under MUD category," said Dr Poudyal. "MUD is a condition of matching gene with other persons. A person's genes match those of only one percent of the population of the entire world," he added.

There is no actual data of patients with bone marrow problems in the country. However, 400 to 600 patients visit Civil Service Hospital for treatment of acute lukemia and other blood cancer cases per year. "Forty to 50 percent patients of blood cancer recover fully while the recovery rate among bone marrow recepients is 70-80 percent," said Dr Poudyal.

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Nepal's sole bone marrow transplant doctor - Republica

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Paul Spagnuolo is working on creating a drug with an avocado compound that targets cancer cells. (Paul Spagnuolo)

A Guelph food science researcher is getting $100,000 from the Ontario Institute for Cancer Research to fund investigations into using an avocado compound as a possible treatment for leukemia.

Paul Spagnuolo discovered that Avocatin B, a compound mainly found in avocado pits can kill leukemia stem cells in 2015.

"Getting funds to do any type of research is a reason to celebrate," said Spagnuolo told CBC News.

The funding will further his research by allowing his lab to use better equipment and collaborate with cancer researchers from the University of Toronto, Princess Margaret Cancer Centre, Ottawa University and McMaster University.

Spagnuolo's lab tested more than 800 natural compounds for their ability to kill leukemia stem cells and discovered Avocatin B was the most potent and only targetedcancer cells.

Avocatin B kills leukemia stem cells by stopping fatty acid oxidation in the cells, a process necessary for the cancer cell to digest fat as a fuel source in order to live and grow.

"Our cells can utilize glucose primarily and some other parts, but leukemia cells are rewired so that if you inhibit the oxidation process, they will die," he said.

Spagnulo and his lab are now looking to develop a way to detect whether or not Avocatin B is circulating in the blood and bone marrow.

Leukemia cells live in the bloodstream or bone marrow, so it's important for the drug to make it to those parts to kill the cancer cells.

"We want to be able to detect our drug inside the blood so that we can understand how we can formulate products better to get our product into the blood," said Spagnuolo.

Moving forward, Spagnuolo's lab will have to report to OICR quarterly, it's a condition of the funding which is spread over two years and has the possibility of renewal for another two years.

"(It's) a lot more intense than I anticipated, but I think the key here is it's very results oriented," said Spagnuolo, "There's no complacency here."

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Pitting avocados against leukemia stem cells - CBC.ca

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New skin begins to regenerate as soon as 4 days after this has been sprayed on patients, compared to skin grafting surgery which may take weeks of pain and possible infections.

Patients who suffer from burn wounds and scars that cant heal on their own only have 1 option: skin graft surgery. This can be very painful, and usually leads to several other complications, and it takes forever to actually heal.

However, RenovaCare has developed a new breakthrough piece of tech: CellMist, a gun that sprays stem cells into a patients burn wound, effectively allowing healthy skin to grow out of it.

It works literally like we described it as. Within 90 minutes of a patient being brought to the emergency room, they stem cells are isolated, processed, put in a liquid suspension, and then loaded into the CellMist gun. CellMist then gently sprays the stem cells onto the patients burn wound.

Tests conducted in Europe and the US have shown that new skin begins to regenerate as soon as 4 days after its been sprayed on patients, compared to skin grafting surgery which may take weeks of pain and possible infections.

Source: Next Big Future

So far, CellMist has only been used to treat second degree burns. However, evidence has shown that it can be used for other skin wounds and skin disorders. They dont think itll work for third degree burns though, as this kind of burn wound has damaged the entire epidermis and dermis levels. CellMist isnt advanced enough to heal such a deep burn wound, and victims would unfortunately have to stick to more traditional methods of treatment. First degree burn wounds on the other hand only barely touch the epidermis, meaning it can still heal on its own, thus not needing such an expensive piece of technology.

It is good to note that even though there is evidence that it might be able to heal things other than burns, CellMist wasnt built to regenerate skin lost from other kinds of injuries or diseases. Its also pretty limited, because as we stated earlier, it should be used immediately after the burn incident has occurred, or else it wont work.

Its still a pretty handy invention. The reason why skin grafting is so risky is because it involves cutting the skin open and leaving it open for 3-4 weeks. This means that nasty bacteria and fungi can easily get into the open wound within that time, causing several infections and complications.

Source: Next Big Future

With CellMist, however, simply involves extracting a thin layer of the patients healthy skin and stem cells and turning it into a spray, and then distributing the stem cells into the burn wound evenly, without damaging other healthy skin cells. The healing time only takes a few days, so there is little chance for an infection to occur if treated properly. And since the patients own skin cells are used in the process, the regenerated skin looks much more natural, with only little scarring. The stem cells grow into fully functioning layers of skin, from the dermis, to the epidermis, to even blood vessels.

Hopefully, this cool new invention will make way for other forms of stem cell treatments for the reconstruction of other organs, like ones heart and kidneys.

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This Stem Cell Gun Helps Burn Victims Grow New Skin Faster - GineersNow (press release) (registration) (blog)

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Intellia Therapeutics is teaming up with Italys San Raffaele University and Research Hospital to develop engineered T-cell therapies for hard-to-treat cancers. The 3-year research collaboration, option, and license agreement will aim to leverage Intellias CRISPR/Cas9 genome-editing platform to generate improved T-cell therapies for both hematologic and solid tumors. The agreement includes options and licenses to key technologies developed at San Raffaele for producing engineered cell therapies.

The collaboration represents the first external partnership for Intellias eXtellia division, which it set up in January 2016 to focus on applying the CRISPR/Cas9 genome-editing platform for applications in immuno-oncology and autoimmune and inflammatory disease indications.

Chiara Bonini, M.D., head of San Raffaeles Experimental Hematology Unit and deputy director of the Division of Immunology, Transplantation, and Infectious Diseases, will lead the scientific work at San Raffaele. Through this collaboration, eXtellia aims to apply CRISPR/Cas9 genome editing in a multifaceted way to modulate the fundamental properties of engineered immune cells and amplify their anticancer properties far beyond current applications, said Andrew Schiermeier, Ph.D., svp at eXtellia. San Raffaele and Dr. Bonini are recognized globally as leaders in cell therapy and immuno-oncology, with excellent track records in translating innovative research into approved therapies."

Intellia is developing a series of ex vivo and in vivo genome-editing programs in-house and in partnership with industry. The firms preclinical in vivo pipeline is focused on using lipid nanoparticles to target the liver and is headed by a Regeneron-partnered program against transthyretin amyloidosis (ATTR). The in vivo pipeline also includes programs targeting alpha-1 antitrypsin deficiency (AATD), hepatitis B virus (HBV), and inborn errors of metabolism (IEMs). Regeneron and Intellia inked their licensing and collaboration agreement to develop CRISPR-based products for up to 10 targetsin 2016.

During late 2014, Intellia and Novartis inked a strategic collaboration and licensing agreement focused on developing ex vivo CRISPR/Cas9-based chimeric antigen receptor (CAR) T cells and hematopoietic stem cells (HSCs). Under the terms of the agreement, Novartis has exclusive rights to develop all programs focused on engineered CAR T cells. Novartis and Intellia are jointly developing multiple HSC programs, and Intellia will in addition develop its own proprietary internal HSC pipeline.

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Intellia, San Raffaele Partner on CRISPR/Cas9-Edited Anticancer T ... - Genetic Engineering & Biotechnology News

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June 06, 2017 08:30 ET | Source: CRISPR Therapeutics AG

BASEL, Switzerland and CAMBRIDGE, Mass. and GOSSELIES, Belgium, June 06, 2017 (GLOBE NEWSWIRE) -- CRISPR Therapeutics AG (NASDAQ:CRSP), a leader in gene-editing based therapeutics, and MaSTherCell SA, a full service contract development and manufacturing organization (CDMO), wholly-owned subsidiary of Orgenesis Inc. (OTCQB:ORGS), today announced the signing of an agreement to develop and manufacture allogeneic CAR-T therapies.

MaSTherCell will be responsible for the development and cGMP manufacturing of CTX101 for use in clinical studies. CTX101 is an allogeneic CAR T-cell therapy currently in development by CRISPR Therapeutics for the treatment of CD19 positive malignancies. The program utilizes CRISPRs proprietary gene-editing technology to make targeted modifications to the T-cell, thereby enabling an allogeneic, or off-the-shelf, product that is applicable to a broader patient population and addresses the challenges of the current generation of autologous therapies.

The signing of this agreement represents an important milestone for CRISPR Therapeutics as it not only demonstrates our progress with CTX101, but also lays the foundation for our broader activities and emerging pipeline in the allogeneic cell therapy field, said Jon Terrett, Head of Immuno-Oncology Research and Translation at CRISPR Therapeutics.

Were really excited to have initiated work with MaSTherCell. Their market-leading capabilities and deeply relevant experience stood out as we looked for a partner to help accelerate our pre-clinical programs towards the clinic in both the US and Europe, added Samarth Kulkarni, President and Chief Business Officer at CRISPR Therapeutics.

MaSTherCell provides process optimization and manufacturing services to cell therapy organizations. It has quickly built the most extensive experience in the field and is focused on developing solutions to the industrialization challenges facing the cell therapy sector.

We are looking forward to working with CRISPR Therapeutics. They have made significant progress to date with multiplexed gene editing and this provides a solid platform upon which we can bring to bear MaSTherCells significant experience in the manufacturing of allogeneic cell therapies, said Denis Bedoret, General Manager at MaSTherCell.

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 Basel, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts. For more information, please visit http://www.crisprtx.com.

About MaSTherCell

MaSTherCell is a dynamic and global Contract Development and Manufacturing Organization (CDMO) on a mission to deliver optimized process industrialization capacities to cell therapy organizations, and speed up the arrival of their therapies onto the market. The company is the subsidiary of Orgenesis Inc. (OTCQB:ORGS), a cell therapy and regenerative medicine company that is committed to developing a cure for Type 1 diabetes. The heart of MaSTherCell is a team of more than 80 highly dedicated experts combining strong experience in cGMP cell therapy manufacturing with a technology-focused approach and a substantial knowledge of the industry. From technology selection to business modeling, GMP manufacturing, process development, quality management and assay development, MaSTherCells teams are fully committed to helping their clients fulfill their objective of providing sustainable and affordable therapies to their patients. The company operates in a validated and flexible facility located in the strategic center of Europe within the Walloon healthcare cluster, Biowin. For more information, please visit http://www.masthercell.com.

About Orgenesis

Orgenesis is a vertically-integrated biopharmaceutical company. The Companys MaSTherCell subsidiary is a global Contract Development and Manufacturing Organization (CDMO) delivering optimized process industrialization capacities to cell therapy companies, and speeding up the arrival of these therapies onto the market. Orgenesis is also developing its own proprietary cell therapies through its subsidiary Orgenesis Ltd., utilizing its proprietary process of Transdifferentiation (or cell reprogramming), whereby an adult cell is converted into another type of cell, such as reprogramming human liver cells into glucose-responsive, fully functional, insulin producing cells, which have the potential to provide a practical cure for insulin dependent diabetes. For more information, visit http://www.orgenesis.com.

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CRISPR Therapeutics and MaSTherCell SA sign service agreement ... - GlobeNewswire (press release)

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On May 12, Sam and Taylor Sabky received heartbreaking news: Their son, Purnell, was diagnosed with Niemann-Pick TypeA, a rare and fatalgenetic disease with no treatment options. Most children with Niemann-Pick Type A die in early infancy.

When we left that appointment, essentially we left with no hope, Sam said. It was kind of a death sentence.

But after learning that research into treatment options is further along than they initially thought and that the biggest obstacle standing in the way is funding the Sabkys decided to create a GoFundMe, with 100 percent of the proceeds going toward funding research into gene therapy, the most promising treatment option for Niemann-Pick Type A.

The campaign went viral, and theyve managed to raise more than $314,000in 10 days, with agoal of $750,000 raised by the end of June. Now, theyre not feeling hopeless anymore.

We have so many people working on his behalf, on our behalf, in many unexpected places, and were so appreciative to have the support of everyone and the response that weve received, Taylor said. I mean, it really makes this feel like its possible and gives us hope and renews your faith in humanity.

Niemann-Pick Type A is a lysosomal storage disease, a type of disease that occurs when toxic materials build up in the cells, as a result of enzyme deficiencies. With Niemann-Pick Type A, symptoms include enlargement of the liver and spleen, and failure to gain weight.

Typically, children diagnosed with Niemann-Pick Type A have a plateau period, which begins around 8or 9months and lasts until theyare between 18-20 months old. During this time, children appear to be interacting normally and possibly even learning some new skills.

Often, after that time, theyll have progressive neurological deterioration, where they really have a hard time interacting, said Dr. Melissa Wasserstein, a Niemann-Pick specialistat the Childrens Hospital at Montefiore in New York City.They lose their focus. Theyre progressively weaker.

Most children with Niemann-Pick Type A die between 2and 3 years of age, according to Wasserstein.

The Sabky family first learned of potential treatment options when they connected with Steve Lafoon. Lafoon is president of the Wylder Nation Foundation, an Arizona-based organization dedicated to accelerating the discovery and development of treatment options for Niemann-Pick Type A.

Lafoons son, Wylder, was diagnosed with Niemann-Pick Type A in 2009 and passed away in 2012. Lafoon said it was difficult for him to grasp not having any treatment options for Wylder, and thats part of what inspired him to start the foundation.

When youre given a diagnosis, and the only option youre told is to go home and enjoy the short amount of time youre going to have, its just something I had a really hard time getting my head around, not having any options, hesaid. So that was our whole premise. We feel that everybody deserves some sort of option moving forward.

According to Lafoon, the most promising treatment option is gene therapy, which is under research at University of California, San Fransiscos Bankiewicz Lab.In order to complete their research and send the treatment option to a clinical trial, the lab has to first build a clinical vector.

[The vector] is basically just a harmless virus that doses a healthy gene to Purnells brain, pretty much replacing the malfunctioning one, Sam said.

But funding is preventing this step from moving forward. The Wylder Nation Foundation has been providing funding to the lab, but researchers need more in order to build the vector. All of the donations to the Sabkys GoFundMe page will go to the Wylder Nation Foundation, which will use it to continue financing the labs research. And since the treatment still has to be approved by the FDA, timing, Sam says, iscritical.

The timing really could line up, which is just so fantastic, but its really a matter of, as soon as the funds are available for that vector, its going to go right to the lab, and theyre going to order and build the vector, and then we can get started, Sam said.

The Sabkys have been overwhelmed by the support theyve received a wealth of different sources. Theyve found strength within the Niemann-Pick Type A community, who have shared Purnells story, as well as from Taylors students at Boston International High School in Dorchester, who have made signs and posters with words of encouragement and inspirational messages for Purnell.

We have everyone in our corner rooting on a cure for this disease, Taylor said.

In the meantime, theyre enjoying every second they have with Purnell, planning vacations and attending weddings, to create lasting memories with their son.

The only thing that has really changed is that when we sit down, were really soaking in the time and being so much more appreciative of him, of the time we have with him, Sam said. Something like this puts stuff in perspective, so we have a lot more gratitude.

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A Roslindale family has raised over $310000 to fund life-saving research for their 1-year-old son - Boston.com

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As a third grade teacher at Ottawa Elementary School, Kelly Gianotti teaches students many important life lessons along with reading and math.

The most important lesson she has instructed was taught by example: how to save the life of a blood cancer patient.

Gianotti donated her stem cells in 2013 to help save the life of a blood cancer patient. The patient was in need of a bone marrow stem cell transplant and had no donor match in her family.

I had seen a flier at a local gym for a high school student who was looking for a match. That intrigued me. I went online to register, Gianotti said.

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A year later Gianotti learned she was a potential match, but not for the high school patient. She went through more testing and did the outpatient donation procedure.

Gianotti later found out her donation assisted MaryAnn Hastings, who lived near Boston, Mass. The two chatted via e-mail and were able to meet in 2016, when Gianotti traveled to Boston.

The lady I donated for died last February of a different type of cancer. I wanted to honor her and spread the word, Gianotti said, adding that Hastings family indicated she was able to give Hastings three extra years of life with her donation.

The donation experience motivated Gianotti to host the first DKMS bone marrow registration drive through Chippewa Valley Schools district. DKMS is an international nonprofit organization dedicated to the fight against blood cancer and blood disorders, according to its website.

The goal of the drive is to help register potential donors. It will be held Tuesday from 4 p.m. to 9 p.m. at Cheyenne Elementary School in Macomb Township. Gianotti said she hopes to register between 100 and 200 potential donors.

Requirements to join the bone marrow registry are that the donor be in good health and between the ages of 18 and 55. The process involves filling out a form, understanding the donation methods and swabbing the inside of each cheek for 30 seconds with a cotton swab. Donors swab their cheeks in a circular motion.

There is no cost to register, although donations are accepted. The donations assist DKMS in covering the $65 registration processing fee.

According to DKMS, 70 percent of people suffering from blood-related illnesses rely on donors other than their families.

If selected as a match for a patient, there are two different methods of donation, according to the DKMS website.

According to the DKMS website, a donation method used in about 25 percent of cases is a one or two hour surgical procedure performed under anesthesia to collect marrow cells from the back of the pelvic bone using a syringe.

To obtain more information about the drive or to make a monetary donation, visit fb.com/cvsgetsswabbed. Those who want to join the bone marrow registry but are unable to attend the June 6 drive can register at dkms.org.

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Chippewa Valley Schools hosts bone marrow registration drive - The Macomb Daily

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COUNTLESS lives across the world could be saved by an Oxfordshire familys appeal to find a bone marrow donor for their little boy.

Two-year-old Alastair Ally Kim has Chronic Granulomatous Disorder (CGD), a life-threatening condition.

He has now become the fourth person in the world to start an experimental gene therapy course at Great Ormond Street Hospital.

In the meantime, his parents have spearheaded 200 international donor drives to find their son a match, signing up 7,000 would-be donors in the process - some of whom have since been matched with other patients.

Father Andrew Kim, 37, of Hinton Waldrist near Longworth, said: We want to use whatever momentum Allys story has to help someone else. We know that matches have come through our drives for other people. Its awesome that someone will benefit from all this.

On Thursday, May 25 family friend Cathy Oliveira organised a drive at the Oxford Universitys Old Road research building, signing up 80 staff members in a day.

Ms Oliveira said: When everything happened with Ally I wanted to show support in any way we could; this is directly beneficial not just for Ally but for others.

Allys CGD means his immune system is compromised and the tiniest infection could leave him seriously ill.

His only chance of a permanent cure is a bone marrow stem cell donation, with a match likely to be of Korean or East Asian origin.

In April the youngster and mum Judy Kim, 36, an Oxford University researcher, travelled to London for him to begin a pioneering new gene therapy treatment.

After a week of chemotherapy to wipe out Allys immune system, cells taken from him are modified in a lab and re-introduced to correct the disorder.

Mr Kim said: Bone marrow would give him back 100 per cent functionality and gene therapy is 10 to 15 per cent; its enough to live in the real world, and not be scared he will die every time he gets an infection.

It has been a roller-coaster of a year, but theres nothing to do but move forward. We are really excited at the thought of him being able to come home this summer.

Blood cancer charity DKMS supported last weeks donor drive in Oxford.

Senior donor recruitment manager Joe Hallet said: Around 30 per cent of patients in need of a blood stem cell donor will find a matching donor within their own family.

The remaining 70 per cent, like Ally, will need to find an unrelated donor to have a second chance of life, so events like these are crucial.

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Donor appeal for poorly toddler 'may have saved other lives' - Bicester Advertiser

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Multiple myeloma is cancer that involves our bone marrow with a specific cell called a plasma cell that patients can develop. Most patients will need a bone marrow transplant.

Patients needing bone marrow transplants dont have to travel far to receive this potentially life-saving transplant.

The actual day of the diagnosis was November 18th of 2015 and it was a diagnosis for multiple myeloma, said Steven Simpson.

Simpson was ready to fight from that day on. He learned from Dr. Kelly McCaul, the director of Avera Hematology Transplant Program, that he would need a bone marrow transplant.

There are many different types of transplant that we do. Theres basically an autologous transplant where patients would be their own donors for their stem cells and then theres allogenic transplant which are some sort of donor process. And so Steve has multiple myeloma. We would normally look at autologous transplant as the preferred pathway for patients with that disease, said Dr. McCaul.

Weve never had to leave anywhere other than here. This is it, said Simpson.

Simpson and his immediate family live no further than 20 minutes away from Avera McKenna so getting the transplant elsewhere was out of the question. But that didnt come without resistance from his insurance company.

Youre asking somebody to go three or four hours out of the way minimum for a period of time that could last anywhere from a week to whatever the process is. You lose your doctors. You lose the ability to have any local family support there as you need them and you dont really know what youre getting into. You just know what youre told, said Simpson.

Simpson and his insurance company worked together and was able to stay at Avera for his transplant.

I came in the day before scheduled for the transplant but left three hours after the transplant because I didnt have any reactions. Plus, we all knew that I had somebody available to watch me 24/7 for the period of time that we would have. The fact that you have your doctors here, your oncologist, your lab people, your nursing staff, everybodys here. They know who you are, said Simpson.

17 years ago when I first looked at this program one of the big things I looked at was the need in the community and it was felt from my perspective, and obviously Avera, that our need in the community was high. And it allows patients to stay within the community, close to family members, without having to drive four, five hours away, said Dr. McCaul.

Today, Simpson is well on his way to feeling like his old self, something he credits to staying close to home for his transplant.

For more information just call 877-AT-AVERA

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Baldness is an accepted part of the aging process for some, and a source of distress for others. Hair loss affects millions of men and women, yet despite decades of research, a cure is still not available. Just how close are we to finding a magic bullet for baldness? Medical News Today take a look at the evidence.

Androgenetic alopecia - which is more commonly known as male pattern baldness and female pattern baldness - is the most common type of hair loss, affecting around 30 million women and 50 million men across the United States.

In men, hair loss begins above both temples and recedes over time to form an "M" shape. Hair also tends to thin at the crown and may progress to partial or complete baldness. In women, the hairline does not recede and rarely results in total baldness, but the hair does usually become thinner all over the head.

Male pattern baldness is hereditary and may be linked to male sex hormones. Male hair loss can start as early as during adolescence. It affects two thirds of men by age 35, and around 85 percent of men by the age of 50.

The causes of female pattern baldness are unclear. However, hair loss happens most frequently in women after menopause, which indicates that the condition may be associated with decreasing female hormones.

With androgenetic alopecia affecting so many people, a permanent cure would not only lessen anxiety for a significant percentage of the population, but it would also prove financially advantageous to the pharmaceutical company responsible for the discovery.

Hair is made up of the hair follicle (a pocket in the skin that anchors each hair) and the shaft (the visible fiber above the scalp). In the hair bulb, located at the base of the follicle, cells divide and grow to produce the hair shaft, which is made from a protein called keratin. Papilla that surround the bulb contain tiny blood vessels that nourish the hair follicles and deliver hormones to regulate the growth and structure of the hair.

Hair follicles, much like all cells, have cycles. A natural part of the cycle involves shedding around 50 to 100 hairs per day.

Each follicle produces hair for 2 to 6 years and then takes a break for several months. While the hair follicle is in its rest phase, the hair falls out. There are around 100,000 follicles on the scalp, but because each follicle rests at a different time and others produce hairs, hair loss is usually unnoticeable. More noticeable hair loss occurs when there is a disruption to the growth and shedding cycle, or if the hair follicle is obliterated and replaced with scar tissue.

Scientists now understand that pattern baldness occurs through a phenomenon known as miniaturization. Some hair follicles appear to be genetically oversensitive to the actions of dihydrotestosterone (DHT), which is a hormone that is converted from testosterone with the help of an enzyme held in the follicle's oil glands.

DHT binds to receptors in the hair follicles and shrinks them, making them progressively smaller. Over time, the follicles produce thinner hairs, and they grow for a shorter time than normal. Eventually, the follicle no longer produces hair, leaving the area bald.

Currently, there are few available treatment options to halt or reverse miniaturization. Most hair loss treatments only manage hair loss, rather than being a permanent solution.

The only two drugs approved by the U.S. Food and Drug Administration (FDA) to treat hair loss are minoxidil (Rogaine) and finasteride (Propecia).

Minoxidil's use for pattern baldness was discovered by accident. Minoxidil was widely used to treat high blood pressure, but researchers found that one of drug's side effects was hair growth in unexpected areas.

Minoxidil lotion is applied to the scalp and may work by increasing blood flow, and therefore nourishment, to the hair follicles. The American Hair Loss Association say that most experts agree that Minoxidil is "a relatively marginally effective drug in the fight against hair loss."

The treatment has zero effect on the hormonal process of hair loss, and its benefits are temporary. Hair loss continues if usage is discontinued.

Finasteride's side effects of hair growth were stumbled upon during the development of a drug to treat enlarged prostate glands.

Finasteride inhibits type II 5-alpha-reductase, which is the enzyme responsible for converting testosterone into the more potent androgen DHT. DHT levels are reported to be reduced by 60 percent when the drug is taken, which prevents the susceptible follicles from being affected by the hormone and returning their normal size.

This treatment does not work in women, and its effect only remains for as long as it is taken.

Dutasteride (Avodart) is used to treat prostatic enlargement. While the FDA has not approved the drug to treat hair loss, physicians sometimes prescribe dutasteride off-label for male pattern baldness.

Dutasteride works similarly to finasteride, but it may be more effective. Like finasteride, dutasteride inhibits the activity of type II 5-alpha reductase. However, dutasteride additionally inhibits type I of the enzyme. Blocking both types of the enzyme lowers DHT even more and reduces the risk of damage to hair follicles.

This drug faces the same limitations as finasteride, meaning that it only works if taken daily and might become less effective over time.

These therapies may slow down or prevent further hair loss, and they could stimulate regrowth from follicles that have been dormant but still viable. However, they can do little for follicles that have already become inactive. Using them at an earlier stage of hair loss will see more favorable results.

Hair transplantation involves harvesting follicles from the back of the head that are DHT resistant and transplanting them to bald areas. A surgeon will remove minuscule plugs of skin that contain a few hairs and implant the plugs where the follicles are inactive. Around 15 percent of hairs emerge from the follicle as a single hair, and 15 percent grow in groups of four or five hairs.

At the end of the procedure, the person will still have the same amount of hair - it will just be distributed more evenly around the scalp. Treating hair loss through surgical procedure can be painful and expensive. There is also a risk of scarring and infection.

Low-level laser therapy (LLLT) is a form of light and heat treatment. LLLT has been shown to stimulate hair growth in both men and women. Researchers hypothesize that the main mechanisms involved in the process is the stimulation of epidermal stem cells in the follicle and shifting the follicle back into the growth phase of the cycle.

Existing medicines for treating hair loss have limited effectiveness and require ongoing use for the benefits of the treatment to continue.

Researchers continue to strive for the holy grail of hair loss cures by trying to gain a better understanding of how the hair growth cycle is controlled. Rather than treating the symptoms of hair loss, scientists aim to target the cause, which, in turn, may yield fewer side effects. Recently, there have been numerous discoveries in the hair loss arena that may lead to new promising treatments.

Researchers from University of Texas (UT) Southwestern Medical Center in Dallas have identified a protein called KROX20, which switches on cells in the skin and tells them to become hair. Furthermore, these hair precursor cells then go on to produce a protein called stem cell factor (SCF), which plays a critical role in hair pigmentation.

When the SCF gene was deleted in the hair precursor cells in mice, they grew gray hair that turned white with age. Moreover, when the KROX20-producing cells were removed, the hair ceased growing, and the mice became bald.

"With this knowledge, we hope in the future to create a topical compound or to safely deliver the necessary gene to hair follicles to correct these cosmetic problems," said Dr. Lu Le, associate professor of dermatology at UT Southwestern.

Future work by the team will focus on finding out whether KROX20 and the SCF gene stop functioning properly and lead to male pattern baldness.

A study led by the University Edinburgh in the United Kingdom discovered 287 genetic regions involved in male pattern baldness. Many of the genes that the researchers identified were linked with hair structure and development.

"We identified hundreds of new genetic signals," said Saskia Hagenaars, a Ph.D. student from the University of Edinburgh's Centre for Cognitive Ageing and Cognitive Epidemiology. "It was interesting to find that many of the genetics signals for male pattern baldness came from the X chromosome, which men inherit from their mothers."

Not only could the team's findings help to predict a man's likelihood of experiencing severe hair loss, but they could also provide new targets for drug developments to treat baldness.

University of California-San Francisco (UCSF) researchers reported that defects in a type of immune cell called Tregs - which are usually associated with controlling inflammation - might be responsible for a different kind of hair loss: alopecia areata. They say that Tregs may also play a role in male pattern baldness.

In a mouse model, Michael Rosenblum, Ph.D., an assistant professor of dermatology at UCSF, and colleagues found that Tregs trigger stem cells in the skin, which promote healthy hair. Without partnering up with Tregs, the stem cells are unable to regenerate hair follicles, and this leads to hair loss.

"It's as if the skin stem cells and Tregs have co-evolved, so that the Tregs not only guard the stem cells against inflammation but also take part in their regenerative work," explained Prof. Rosenblum. "Now the stem cells rely on the Tregs completely to know when it's time to start regenerating."

Hair growth can be restored by inhibiting the Janus kinase (JAK) family of enzymes that are located in hair follicles, according to investigators from Columbia University Medical Center (CUMC) in New York City, NY.

Tests with mouse and human hair follicles showed that applying JAK inhibitors directly to the skin promoted "rapid and robust hair growth." Two JAK inhibitors that are approved by the FDA include ruxolitinib (for the treatment of blood diseases), and tofacitini (for the treatment of rheumatoid arthritis).

In a small clinical trial, Angela M. Christiano, Ph.D. - the Richard and Mildred Rhodebeck Professor of Dermatology and professor of genetics and development at CUMC - reported that treating moderate to severe alopecia areata with ruxolitinib triggered an average hair regrowth of 92 percent.

Prof. Christiano and team plan to expand their studies to include testing JAK inhibitors in other conditions and pattern baldness. "We expect JAK inhibitors to have widespread utility across many forms of hair loss based on their mechanism of action in both the hair follicle and immune cells," she added.

Researchers from the Sanford-Burnham Medical Research Institute in San Diego, CA, developed a technique to generate new hair using pluripotent stem cells. This method would provide an unlimited source of cells without being limited to transplanting follicles from one part of the head to another.

Alexey Terskikh, Ph.D., associate professor in the Development, Aging, and Regeneration Program at Sanford-Burnham, and collaborators coaxed human pluripotent stem cells to become dermal papilla cells.

"We developed a protocol to drive human pluripotent stem cells to differentiate into dermal papilla cells and confirmed their ability to induce hair growth when transplanted into mice," said Prof. Terskikh. The next step in their research is "to transplant human dermal papilla cells derived from human pluripotent stem cells back into human subjects."

Although giant strides to cure baldness are being made in laboratories globally, research is ongoing and the wait for a permanent solution continues.

Excerpt from:
Baldness: How close are we to a cure? - Medical News Today

Recommendation and review posted by Bethany Smith

CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is the basis for a revolutionary genome-editing technology that allows researchers to make very precise modifications to DNA.

The implications are enormous not only for the treatment of disease, but also for genetic engineering and scientific research more broadly.

It's no surprise, then, that CRISPR has been making the news, especially over the last few years, as its applications have become increasingly appreciated and developed. In 2015, the journal Science named CRISPR the breakthrough of the year; earlier this year, Emmanuelle Charpentier and Jennifer Doudna received the 2017 Japan Prize for their role in inventing the technology just one of the latest in a string of honors. CRISPR seems to be everywhere, from headlines to classrooms.

So surely everyone understands exactly what CRISPR is and how it works?

Or not. That's one reason a new video, produced by Wired magazine, is worth a look. The video features Dr. Neville Sanjana, an assistant professor at New York University and a member of the New York Genome Center, explaining CRISPR five ways: to a child of 7; a high school student; a college student; a graduate student; and an expert in the field. The conversation ranges from genomes to allergies, from ethics to the value of basic research.

Besides offering a valuable introduction to CRISPR, the video illustrates how understanding can evolve from a relatively short description to a dialogue with more nuance. Research finds that we indeed explain science differently to children than to adults, and Sanjana's explanations showcase some of that range.

The video won't transform your genome, but it might make a helpful edit to your understanding.

Tania Lombrozo is a psychology professor at the University of California, Berkeley. She writes about psychology, cognitive science and philosophy, with occasional forays into parenting and veganism. You can keep up with more of what she is thinking on Twitter: @TaniaLombrozo

Originally posted here:

Five Different Ways To Explain CRISPR : 13.7: Cosmos And Culture ... - NPR

Recommendation and review posted by sam

In case you havent already heard of CRISPR-Cas9, it is the revolutionary gene-editing technology, discovered just a few years ago, that allows scientists to edit the DNA of any species with an unprecedented precision and efficiency. Today, thousands of researchers around the world are doing experiments with CRISPR, in the hope to cure us from genetic diseases and even deliver us designer babies. The first clinical trial to employ CRISPR-Cas9 is now underway in China, hoping to fight targeted cancers with modified immune cells.

The gene-editing method is based on the protective mechanism of bacteria against viruses. An RNA molecule carries segments of DNA from a previously encountered virus together with an enzyme (Cas9). Once the molecule encounters that same sequence of DNA, the enzyme gets activated and cuts it out. Researchers discovered that they can use this system to cut any DNA sequence at a precisely chosen location.

While the tool is touted for its precision, it is far from error free. Mutations do occur around the areas where the DNA has been cut and needs to be repaired. And sometimes CRISPR may hit unintended parts of the genome. Computer algorithms identify the most likely areas for these off-target mutations, which are later examined by researchers for deletions and insertions. However, whole-genome sequencing (WGS) - examining the entire DNA of living animals that had undergone gene editing - hadn't been done.

In a recently published study in the journal Nature Methods, titled Unexpected mutations after CRISPRCas9 editing in vivo scientists used whole-genome sequencing to study the mutations that had occurred in the DNA of mice that had undergone CRISPR gene editing.

Genetic Engineering and Biotechnology News reports that the investigators were able to determine that CRISPR had successfully corrected a gene that causes blindness, but found that the genomes of two independent gene therapy recipients had sustained more than 1500 single-nucleotide mutations and more than 100 larger deletions and insertions. None of these DNA mutations were predicted by computer algorithms that are widely used by researchers to look for off-target effects.

Co-author of the study Vinit Mahajan, M.D., Ph.D. said:

"We're still upbeat about CRISPR, we're physicians, and we know that every new therapy has some potential side effectsbut we need to be aware of what they are."

The authors are encouraging scientists to use the WGS method to determine all off-target effects of their CRISPR experiments.

In the last few days, however, some scientists have raised concerns about the validity of the study, questioning its methodology. Dr Gaetan Burgio, Group leader and head of the transgenesis facility at the Australian National University said in a Journal club review of the paper:

The claims over this paper are unsurprising as Cas9 enzyme could remain in the cells for days and create random indels in the genome. However, the main issues for me resides in the overestimation of the number of off target effects due to the lack of rigor in the experimental design to detect these unexpected mutations. In short my main point is these unintended mutation are likely to have preexisted prior to the injection of CRISPR system.

One thing is sure there is a lot more work to be done to ensure the safety of the CRISPR/Cas9 technology.

Photo Credit:Pixabay

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CRISPR May Cause Hundreds of Unintended Mutations Into the ... - Big Think

Recommendation and review posted by sam