Genetherapy

Lipid nanoparticles for gene therapy — ScienceDaily – Science Daily

February 15th, 2017

25 years have passed since the publication of the first work on solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) as a system for delivering drugs. So the European Journal of Pharmaceutics and Biopharmaceutics has prepared a special edition for which it asked the PharmaNanoGene group of the UPV/EHU-University of the Basque Country to produce a piece of work reviewing the application of SLNs and NLCs in gene therapy since the group's significant contributions made in this area have been included in various international scientific publications.

Lipid nanoparticles (SLNs and NLCs) are regarded as highly promising systems for delivering nucleic acids in gene therapy. Until now, viral systems have been the most effective method for delivering genetic matter but they pose significant safety problems. "Non-viral vectors, including SLNs and NLCs, are less effective but much safer even though their effectiveness has increased significantly in recent years," pointed out Alicia Rodrguez, Mara ngeles Solins and Ana del Pozo, authors of the article published in the European Journal of Pharmaceutics and Biopharmaceutics.

This review article describes these systems and their main advantages in gene therapy, such as their capacity to protect the gene material against degradation, to facilitate cell and nucleus internalisation and to boost the transfection process. "What is more, the nanoparticles are made up of biocompatible, biodegradable materials, they are easy to produce on a large scale, they can be sterilised and freeze-dried and are very stable both in biological fluids and in storage," explained the researchers.

This review also includes the main diseases in which lipid nanoparticles are being applied, generally on the preclinical level: degenerative diseases of the retina, infectious diseases, metabolic disorders, and cancer, among others. "At PharmaNanoGene we are working on the design and evaluation of SLNs for treating some of these diseases using gene therapy. We are studying the relationship between formulation factors and the processes involving the intracellular internalisation and disposition of the genetic material that condition the effectiveness of the vectors and which is essential in the optimisation process, and for the first time we have demonstrated the capacity of SLNs to induce the synthesis of a protein following their intravenous administration in mice," they stressed.

The publication also includes other pieces of work by this UPV/EHU research group on the application of SLNs in the treatment of rare diseases, such as chromosome-X-linked juvenile retinoschisis, a disorder in which the retina becomes destructured due to a deficiency in the protein retinoschisin. "One of the main achievements of our studies in this field has been to demonstrate, also for the first time, the capacity of a non-viral vector to transfect the retina of animals lacking the gene that encodes this protein and partially restore its structure, showing than non-viral gene therapy is a viable, promising therapeutic tool for treating degenerative disorders of the retina," specified the researchers.

The application of SLNs for treating Fabry disease, a serious, multi-system metabolic disorder of a hereditary nature, has also been studied at PharmaNanoGene. "This is a monogenic disease linked to the X-chromosome which is caused by various gene mutations in the gene that encodes the a-galactosidase A (a-Gal A) enzyme. In cell models of this disease we have demonstrated the capacity of SLNs to induce the synthesis of a-Gal A." They have also reviewed the application of lipid nanoparticles to the treatment of infectious diseases: "Our work in this field shows that SLNs with RNA interference are capable of inhibiting a replicon of the hepatitis C virus in vitro, which was used as proof-of-concept of the use of SLN-based vectors as a new therapeutic strategy for treating this infection and others related to it."

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Human genome editing shouldn’t be used for enhancement yet – New Scientist

February 15th, 2017

CRISPR: gene editing made easy

Ella Maru Studio/SPL

By Jessica Hamzelou

While gene editing is already saving lives, for now, the technique shouldnt be used to edit embryos or create changes that will be passed on through the generations. So say the authors of a new report on editing the human genome.

However, such germline editing could be permitted in the future, if properly regulated and with public approval, concludes the report. It was compiled by the Committee on Human Genome Editing, a group of 22 researchers, lawyers and ethicists.

Gene therapy isnt new, but the development of the CRISPR Cas-9 technique has made it much easier to change a genome. The technique enables researchers to specifically target a region of DNA and add or remove genes both a useful tool for research, and a technique that can treat diseases in people.

But gene editing treatments are not without some risk. Theres a chance, for instance, that a therapy will have off-target effects, changing other genes. The risks will depend on the disorder and the treatment, and regulators must weigh up the risks against treatment benefits on a case-by-case basis, the authors say.

The risks are higher when it comes to germline editing. Beyond off-target effects, theres a chance that attempts to perform gene editing on an embryo will create a mosaic of treated and untreated cells. Its the most common problem in mouse studies, says Robin Lovell-Badge of the Francis Crick Institute in London, who co-authored the report.

Lovell-Badge and his colleagues concluded that germline editing could be performed in humans, but only after much more research to minimise the risks and weigh them up against any benefits. Even then, the public must have a say, and any trials must be performed under strict oversight.

The report is also not in favour of gene editing techniques to enhance people, or create designer babies but only for the time being. Its the thing that worries people the most, because it is felt to be unfair, says Lovell-Badge. Its the same as using drugs to cheat.

But the boundary between treatment and enhancement is often blurred. If you were able to lower a persons cholesterol for example, where would the cut-off be? In the future, some aspects of enhancement might be considered acceptable, says Lovell-Badge. We may need to modify aspects of our physiology to adapt to climate change, but thats being speculative, he says. Were not saying it should never be done but not now.

Based on what we already know about genes and health, it might be possible to boost a persons muscle mass, for instance, using gene editing. But for many other features including intelligence hundreds or thousands of genes are involved. Using gene editing to enhance these features isnt currently feasible.

Read more: Why banning CRISPR gene editing would be unnecessarily cautious

Radical Life Extension Is Already Here, But We’re Doing it …

February 13th, 2017

We've already tacked three decades onto the average lifespan of an American, so what's wrong with adding another few decades?

A centenarian riding his bike in Long Beach, California (Reuters).

So far as we know, the last hundred years have been the most radical period of life extension in all of human history. At the turn of the twentieth century, life expectancy for Americans was just over 49 years; by 2010, that number had risen to 78.5 years, mostly on account of improved sanitation and basic medicine. But life extension doesn't always increase our well-being, especially when all that's being extended is decrepitude. There's a reason that Ponce de Leon went searching for the fountain of youth---if it were the fountain of prolonged dementia and arthritis he may not have bothered.

Over the past twenty years, biologists have begun to set their sights on the aging process itself, in part by paying close attention to species like the American Lobster, which, despite living as long as fifty years, doesn't seem to age much at all. Though some of this research has shown promise, it's not as though we're on the brink of developing a magical youth potion. Because aging is so biologically complex, encompassing hundreds of different processes, it's unlikely that any one technique will add decades of youth to our lives. Rather, the best we can hope for is a slow, incremental lengthening of our "youth-span," the alert and active period of our lives.

Not everyone is thrilled by the prospect of radical life extension. As funding for anti-aging research has exploded, bioethicists have expressed alarm, reasoningthat extreme longevity could have disastrous social effects. Some argue that longer life spans will mean stiffer competition for resources, or a wider gap between rich and poor. Others insist that the aging process is important because it gives death a kind of time release effect, which eases us into accepting it. These concerns are well founded. Life spans of several hundred years are bound to be socially disruptive in one way or another; if we're headed in that direction, it's best to start teasing out the difficulties now.

But there is another, deeper argument against life extension---the argument from evolution. Its proponents suggest that we ought to avoid tinkering with any human trait borne of natural selection. Doing so, they argue, could have unforeseen consequences, especially given that natural selection has such a sterling engineering track record. If our bodies grow old and die, the thinking goes, then there must be a good reason, even if we don't understand it yet. Nonsense, says Bennett Foddy, a philosopher (and flash game developer!) from Oxford, who has written extensively about the ethics of life extension. "We think about aging as being a natural human trait, and it is natural, but it's not something that was selected for because it was beneficial to us." Foddy told me. "There is this misconception that everything evolution provides is beneficial to individuals and that's not correct."

Foddy has thought long and hard about the various objections to life extension and, for the most part, has found them wanting. This is our conversation about those objections, and about the exciting new biology of aging.

Foddy: The reason I present it that way, is that there's always this background moral objection in enhancement debates, where a technology is perceived to be new, and by virtue of being new, is depicted as threatening or even strange. That goes for everything from genetic engineering to steroids to cloning and on and on. I think it's always worth contextualizing these things in terms of the normal. So with human cloning it's worth remembering that it's exactly the same as twinning. With steroids, it's worth remembering that in many ways it's not that different from training and exercise, and also that people have been taking testosterone since ancient times. I think this way you can kind of resist the idea that something is wrong just because it's strange.

When you're talking about medicines that help us live longer, it's important to realize how much we've already accomplished. In the last 150 years or so, we've doubled our life span from 40 to 80 years, and that's primarily through the use of things you can characterize as being medical science. In some cases it's clear that we're talking about medical enhancement---vaccines, for instance, or surgical hygiene and sterilization. And then more broadly there are other, non-medical things like the sanitation of the water supply and the pasteurization of milk and cheese. All of these things have saved an enormous amount of life.

It used to be that people would die of an infectious disease; they'd be struck down when they were very young or when they were older and their immune system was weak. Now almost nobody in the first world dies of infectious disease; we've basically managed to completely eradicate infectious disease through medical science. If, at the outset of this process, you asked people if we should develop technologies that would make us live until we're 80 on average instead of until we're 40, people might have expressed these same kind of misgivings that you hear today. They might have said, "Oh no that would be way too long, that would be unnatural, let's not do that."

So, in a way, we shouldn't view it as being extremely strange to develop these medicines, but in another sense we're at a new stage now, because now we're at the forefront of having medicines that actually address the aging process. And that's what I'm interested in talking about---the kinds of medicines that actually slow down the aging process, or at least some of the mechanisms of aging.

Can you explain how senescence, the biological process of aging, is unevenly distributed across species?

Foddy: There are different animals that are affected differently by various processes of aging. In my paper I go into the case of the American Lobster, which lives about as long as a human being. When you dissect one of these lobsters at the end of its life, its body doesn't show much in the way of weakening or wasting like you see in a human body of advanced age. That suggests that aging can evolve differently in different species. Lobsters seem to have evolved an adaptation against the cellular lifespan. There's this phenomenon where the DNA in our cells basically unravel after they've divided a certain amount of times, but lobsters have this enzyme that helps them replenish their telomeres---the caps that hold DNA together.

That's one of the reasons why lobsters don't seem to undergo aging in the same way that we do. Other species give off an antioxidant chemical in their bodies that prevent these oxidizing free radicals in our bodies from breaking us down. That's why doctor's recommend that you have a certain amount of antioxidants---some species are really good at producing those naturally.

There is this idea that when you're evolving you make certain trade-offs. Lobsters and clams don't really move around a lot; their bodies move and grow very slowly and one of the upsides of that is that they've been able to invest their evolutionary chips, so to speak, in resisting the aging process. Human beings, on the other hand, have to move around quite a lot. We have giant brains and we have to be able to run away from saber tooth tigers. As a result we have bodies that burn a lot of calories, and so that's where our chips are invested. It's just a difference in our evolutionary environment and that's why we've evolved to live and die the way we do. But it could have easily not turned out that way---that's the point I really want to make.

What are the current biological limits on our human life span, or our human "youth span," as you call it---the time that we're able to live as young, vibrant, reproducing individuals?

Foddy: The sky is sort of the limit there. There won't be a magic pill that gives us infinite youth, but over time there will probably be different technologies that allow you a few extra years of youth. We think of aging as being a unitary thing, but it's made up of hundreds of different processes. So, one of the different things we think about, for example, is dementia, the state where your brain sort of wastes away. Now, if we discover a way of reversing that process, or slowing that process, that would be one dimension where we no longer age, where our minds will stay youthful for longer. It's also possible that we might be able to find a way of stopping people's muscles from wasting away as they get older.

Nothing is going to be super dramatic, but there will be a point where you'll look back a hundred years and notice that people used to get really kind of feeble and after awhile they weren't capable of really thinking or processing information anymore, and they had to go into a home and they had to be looked after and nursed for a time. And that will seem very old-fashioned and very barbaric, but I very much doubt it will happen at a moment in time where we suddenly realize that some magic pill has exponentially extended our youth. Part of that's because we're not exactly clear what aging is. We've identified a whole range of processes, but there ere still a whole lot of arguments in the scientific community about what is really responsible for aging, and which of the processes are subsidiary to other processes.

Have we glimpsed, even theoretically, ways that we might add to that youth-span. What are the bleeding edge technologies that might allow us to overcome aging?

Foddy: I'm not a scientist, so I don't want to weigh in too heavily on somebody's body of research. We've seen promising results looking at the lobsters and we've seen promising results with antioxidants, even aspirin, but as I said these things are going to be incremental. You meet a lot of people in the scientific community that are true believers and they're expecting a kind of a radical thing. And it's not as though we never have a radical thing in medicine, but what we have more frequently is incremental advances.

Cancer is a great example of the kind of incremental progress I'm talking about. In 1970, your odds of surviving 5 years after you've were diagnosed with certain kinds of cancer were slim; those chances have increased substantially. But we still react to the idea of getting cancer as though it were 1970 because we don't really process incremental changes. Like with chemotherapy, they just change out one or two drugs every year based on trials that show that the new drug is 2 percent more effective than the previous drug. That's constantly going on, but it really isn't announced. Instead, we get the occasional story in the news about a miracle cure for cancer, and it always turns out not to be as good as they had hoped and everyone begins to get disillusioned about science and the value of medical progress. But when you run the comparisons across decades, you see something much more dramatic.

You give an interesting account of how the aging process evolved in humans. You argue that aging is not the result of an optimizing process, but that instead it's a byproduct of an optimizing process. Can you explain why that difference is so important?

Foddy: I should say, first of all, that this is not original to me; this is very well established in evolutionary biology. We have a number of genetic traits that we developed because they were advantageous from the perspective of natural selection---that is, they helped us to survive and reproduce. People that had the gene for that trait had the ability to reproduce more than people that didn't have it. It's easy to imagine that every gene that we have is selected because it gave a positive advantage in this way, but it turns out there are trade-offs. A number of the processes of aging seem to have arisen because our bodies were not doing enough maintenance, because they were busy doing something else. The misconception that people often have is that any trade-off that we have is going to be directly beneficial, directly advantageous. But that's not right.

The second thing to say is that aging usually happens to an organism after it reaches menopause. Things that happen after menopause are much less interesting in terms of evolution, because they have much less of an effect. If I've already reached the age where I can't reproduce, then aging that takes effect at this point in my life is not going to affect whether or not I reproduce. The game is sort of already over for me. As a result, natural selection doesn't tend to weed out genes that take effect after you've reached the age of menopause. So, there is this idea that over time you can amass genes in your genome that have nothing to do with survival or not surviving, because they only activate after you reach a certain age. So, over time, some of these are going to be good genes and some of them are going to be bad. It's going to be this kind of mix, but it's certainly not going to be the case that they're on balance beneficial. We've got hundreds or thousands of genes that don't start to harm us until we reach old age, and those genes are responsible for a lot of what actually constitutes aging. So, in this sense, we think about aging as being a natural human activity or a human trait---and it is natural, but it's not something that was selected because it was beneficial to us. There is this misconception that everything evolution provides has to be beneficial to individuals and that's not correct. "There is this misconception that everything evolution provides has to be beneficial to individuals and that's not correct."

One defense of aging that your paper takes quite seriously is the argument from evolution, which was first put forth by Frances Fukuyama. Fukuyama claims that we should resist the temptation to tinker with any characteristic that we have been given through the process of natural selection. He argues that evolution can be relied upon to produce good results and that we ought not to mess with the fruit of its processes. What's wrong with this view?

Foddy: Fukuyama has this idea that evolution is very complicated, which is true. We don't always understand why we've evolved to be a certain way. Sometimes it looks like something is useful, but in fact it's performing some kind of role that we don't know much about. Fukuyama is also correct that sometimes we interfere with complicated biological systems without really understanding what the effects will be, and that then we wind up with some unwanted effect. That's all true.

The thing that I disagree with him about is his presumption that if we have a trait that's evolved, that it must be beneficial to us in some way, and that we have some good reason for allowing that trait stick around. Now he's not talking strictly about aging; his book discusses all kinds of intervention on the human organism. But, when it comes to aging, his argument can't even succeed on its own merits, because we know for a fact that aging is not the sort of thing that is produced by natural selection in the kind of positive way that he is talking about. He says it's not always easy to do nature one better, but that's not what we're doing when we're combating aging. We're not trying to do nature one better, because nature doesn't care that we grow old and die. This is neglect, evolutionary neglect. We shouldn't think about it as interfering with the sort of complex ecological balance in the way that he's worried about.

Now that's not to say that our current mode of life extension is ideal. Some of the biggest strains on our resources stem from the fact that populations are getting older as birthrate's go down, especially in the first world. Aging societies are spending more and more on nursing, and so I think that it makes sense to pursue a youth-extending medicine that would diminish the number of years that we have to spend in nursing homes. You could imagine us living more like the lobster, where we still live to be about 80-85, but we're alert and active until we drop dead. In that scenario we wouldn't have this giant burden where the state has to support and pay to nurse people that are unable to look after themselves anymore.

Now, it has to be said that the story of medicine and medical progress in the past 50 years has not been heading that way. If anything, we're extending the number of years that we spend needing nursing. We've gotten good at keeping people alive once they're fairly decrepit. And that sort of guarantees that you have the maximum drain on resources, while also producing the kind of minimum amount of human benefit. You get to be 90 years old and your hip goes out, and we give you a massively expensive hip replacement, but we don't do things to prevent your body from wasting away and becoming corroded when you're 20, 30 or 40.

There's this great Greek myth, the myth of Tithonus, that always comes to mind. Tithonus was a mortal who was in love with Eos, the goddess of the dawn. Eos didn't want Tithonus to grow old and die, so she went to Zeus to ask for eternal life, which was granted. But, she forgot to ask for eternal youth, and so Tithonus just gets older and older and more decrepit, and eventually he can't really move, and then finally he turns into a grasshopper in the end. That's sort of the course that we're on with our current approach to medicine and life extension.

Some ethicists have pointed out that death is one of the major forces for equality in the world, and that welfare disparities will be worsened if some people can afford to postpone old age, or avoid it altogether, while others are unable to. What do you say to them?

Foddy: I think that's right. I mean there are concerns whenever we develop any kind of medicine or any kind of technology---the concern that these things are going to widen welfare gaps. The story of industrialization is that the people who could afford the cars and machines and factories in Western countries were able to produce a lot more and generate a lot more wealth than people in poorer agrarian economies. That's a serious issue. It's probably true that if people in the first world were, through some sort of medical intervention, able to live to be 200 years old and people in Bangladesh were still dying at a relatively young age, that would tend to widen the distance in personal wealth.

And look this has already happened. It's already unfair that I will on average live to be 80 and yet, if I were born before some arbitrary date, or in some other place, I would live much less longer. Those things are unfair and it's worth worrying about them, but I don't think the correct response is to hold off on the science. It's better if everybody can eventually get this medicine, because living a long time is not a positional good, it's an absolute good. It would be great if everybody could live to be 150, because that would benefit every single person. It's not a good that benefits you only if other people are worse off. When you have goods like that you should try to develop them and then you should worry separately about making sure that they get delivered to people in poorer areas, whether it's through government aid or massive production.

Another objection to the elimination of aging is this idea that the aging process makes an elderly person's death less painful for the survivors around her, because it gradually forces people to stop relying on her, and forces her to gradually remove herself from society. You call this the argument from psycho-social history.

Foddy: This is Leon Kass' argument. He thinks aging is just fantastic for this reason because it helps us to let go of somebody. And of course it's true that when people grow old, they become less useful to society, and more socially difficult, which places burdens on people. And in a lot of cases we respond to this by cutting them out of our lives, essentially. People get older, they move into a nursing home, and we see them less and less, and then when they finally die everyone's like, "well it was expected." Advanced age sort of helps us prepare emotionally for letting go of people, but it seems to me that it's not good for the person who gets old.

Now, what would the world be like if people dropped dead in good health when they reach a certain age? It would be very sad, but on the upside the person would've had 20 or 30 years of additional integration into society and we would've been able to spend more time with them. I've got to say that I would've enjoyed my grandmother's presence a lot more if she'd been able to run around and to play and work and be part of society in her extremely advanced age.

Nick Bostrom has said that people have fallen victim to a kind of Stockholm syndrome when it comes to aging. The idea being that because aging has always been an insurmountable obstacle for humanity, that we have dignified it more than it deserves, that we contort ourselves logically and rhetorically to defend it precisely because it is so inescapable. Does that sound right to you?

Foddy: Yes, I think that's right, although Nick draws conclusions that are a bit more extreme than I would tend to draw. I think that we do have a tendency to kind of rationalize things that we don't think we can do anything about. This is a perfectly healthy attitude if you really can't do anything about the aging process---it's better to accept it and kind of talk about it as being a natural part of life, not something to rail against or feel bad about. It's something that everybody goes through. Now if it did so happen that we could discover a medicine that completely prevents that process from taking place, we would have to re-evaluate at that stage and realize that we've done some emotional rationalization here and the conditions for it no longer apply. We no longer need to comfort ourselves with the inevitability of death if it's not actually inevitable.

Having said that, death is, in fact, inevitable. Even if we solve every medical problem, you still have a 1 in 1,000 chance of dying every year by some sort of accident. So, on those odds you could probably expect to live to be about 1,000. I don't think it's ever going to be the case that we will live forever. It's not even going to be 1,000. We're probably talking about living to be 120 or 150 or somewhere around there, but to me the idea that we have to accept living to 80 rather than 120 is bizarre given that it's not so long ago that we lived to 40.

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Radical Life Extension Is Already Here, But We're Doing it ...

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Orbital ATK Sues DARPA Over Satellite-Repairing Robots | Inverse – Inverse

February 13th, 2017

Private space technology company Orbital ATK sued the Pentagons Defense Advanced Research Projects Agency (DARPA) last Tuesday over plans to give a rival firm a contract to build satellite-repairing robots for a government-funded mission.

The Virginia-based company filed a complaint with the U.S. District Court for the Eastern District of Virginia, asking court to halt DARPAs work on the Robotic Servicing of Geosynchronous Satellites (RSGS) mission, which would promote and develop robotic satellite repair technology.

DARPA chose rocket manufacturer Space Systems Loral (SSL), which is a subsidiary of Canadas MDA Corp., to award a $15 million contract for building robots for repairing government and commercial satellites.

It clearly demonstrates the success of our strategy to bring the benefits of our commercial business to a broader audience and to grow our business with U.S. government work, Howard Lance, CEO of SSL MDA Holdings, said in a statement last Thursday.

According to Jared Adams, DARPAs chief of media relations, the RSGS public-private effort is a first for DARPA in the space-servicing domain, and DARPAs selection of SSL has been submitted for review by the Defense Departments Under Secretary of Defense for Acquisition, Technology and Logistics.

In the lawsuit, Orbital ATK argued that DARPA intends to give away this technology to a foreign-owned company for that companys sole commercial use. In addition, Orbital ATK said RSGS would waste hundreds of millions of U.S. taxpayer dollars to develop robotic satellite servicing technology for which DARPA has admitted there is no present U.S. government need and that NASA and the U.S. private sector specifically the plaintiffs are already developing.

On the other hand, DARPA says RSGS would lower the risks and costs of operating in orbit.

Servicing on orbit could provide significant cost savings compared to current practices and a major advantage to the security of both commercial and Government space assets, Gordon Roesler, DARPAs program manager for RSGS, said in a statement last Thursday.

RSGS, Orbital ATK argues, directly competes with Orbital ATKs Mission Extension Vehicle, which is in development and provides life extension services to satellites. The company argues this violates the 2010 National Space Policy, which says that the government must refrain from conducting United States government activities that preclude, discourage, or compete with U.S. commercial space activities.

Currently, the Mission Extension Vehicle is backed by at least $200 million from investors, and Orbital ATK had set up a production facility in Northern Virginia. The company planned to launch the Mission Extension Vehicle next year.

In the past, Orbital ATK has worked with the U.S. government and NASA on various space projects. On Monday, the company announced that the U.S. Air Force awarded it a contract to provide support services for a multipurpose satellite.

This isnt the first time DARPA was asked to stop RSGS. Two weeks ago, three Republican members of Congress wrote a letter to the Pentagon saying RSGS violates the National Space Policy because its competing with a private company.

We urge you to promptly review this program to ensure its compliance with the 2010 National Space Policy, the letter to DARPA Acting Director Steven Walker said. As Acting Director, you should stop any further action on RSGS until the review is completed.

Walker replied saying that the commercial systems under development would not be as capable as RSGS, and he reviewed the mission, saying that it complied with the National Space Policy.

DARPA also said that a NASA satellite repair program called Restore-L does not have the same degree of autonomous control as RSGS. Restore-L was also awarded to Space Systems Loral and is planned to launch by 2020.

Photos via Flickr / NASA Johnson

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Orbital ATK Sues DARPA Over Satellite-Repairing Robots | Inverse - Inverse

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What’s in store: Chronos – bestofneworleans.com

February 13th, 2017

Chronos (3200 N. Arnoult Road, Metairie, 504-267-4549; http://www.chronosbhw.com) is locally owned and operated by Drs. Mace Scott and Miguel Aguilera. Scott and Aguilera are daily fixtures at Chronos, as is Dr. Shannon Pickens, a cosmetic dermatologist. All three share the philosophy that mental and physical health require a multi-tiered approach focused on improving well-being through exercise and propernutrition.

Chronos opened in Metairie in 2013. Scott, an ER physician, noticed that many of the patients he treated could have avoided a trip to the hospital if preventive measures had been taken. This, and personally noticing the effects of aging on his own body, motivated him to create a place that addressed these issues.

"We want to help people have a better quality of life," Scott says. "Our goal is to help improve your life, improve your health and improve your overall well-being." Chronos incorporates a medical spa, a day spa and a fitness facility all under one roof. Chronos' mission is to help clients look better, feel healthier and be happier through preventive care.

Mace challenges the belief that having less energy and becoming softer around the middle is a natural part of the aging process. He and his team offer services that minimize the physical effects of aging, such as hormone replacement therapy (HRT), a medical procedure that treats hormone deficiencies associated with perimenopause and post menopause in women and andropause in men. As men and women age, their hormones often have unexpected effects on their moods, energy levels, libidos and bodies. HRT rebalances hormones that were once abundant in youth and can help enhance quality of life.

Chronos also offers a range of cosmetic non-surgical treatments. Fillers like Botox, Restylane and Dysport are available, as well as body sculpting services like Cool Sculpting and BodyFX, non-invasive treatments that can help reduce fatty deposits in problem spots like the thighs, chin and stomach area, with no down time. Chronos also offers state-of-the-art facials like intense pulsed light (IPL) photofacials, Ultherapy, Forma and Fractora treatments, microneedling and HydraFacials, in addition to other spa services such as massages, manicures and pedicures.

Chronos recently launched its EMPower fitness series. EMPower helps participants build up their endurance and strength through a rigorous 60-minute workout that incorporates boxing and interval training while also building core strength. Chronos' fitness center is open daily, and offers boot camp, spinning and yoga classes, as well as one-on-one personal training and individualized workout plans.

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What's in store: Chronos - bestofneworleans.com

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‘Bathroom Bill’ is discrimination – Emporia Gazette

February 13th, 2017

Current House Bill Text ColorSwatch/NoneStrokeStyle/$ID/SolidText ColorSwatch/NoneStrokeStyle/$ID/Solid$ID/NothingText ColorText Color$ID/NothingText ColorText Color2171 defines sex as the physical condition of being male or female, which is determined by a persons chromosomes, and is identified at birth by a persons anatomy as if this is always the case.

In the case of transgender children, this is not the case. This bills wording reveals major biological ignorance and discrimination against a handicap.

Usually, but not always, XY chromosomes produce a boy and XX produces a girl. However, a relatively high rate of babies are born XO: one X chromosome and no second X or a Y. This is called Turner syndrome. I have had at least six Turner syndrome students in my classrooms during my teaching career that I know of. Additional cases when chromosomes fail to separate normally produce individuals who are XXX, XXY, XYY, and XXXY, etc.

The bill states that sex is identified at birth. Not always. If a couple do not already know the sex of their child, the first announcement they await is its a boy or its a girl! But sometimes the delivering physician has to say Ill get back to you on that when the genital anatomy is ambiguous and not clearly either male or female. There are many causes for when the body does not follow normal development. Today, science understands most of them.

In the case of androgen insensitivity, a baby girl appears to have the external and internal anatomy of a girl, until they biopsy the gonadal tissues and discover internal testes! Testes produce testosterone that flows through the bloodstream and body tissues respond to the testosterone by developing male tissues. But in this case, the childs cells lack the receptors and ignore the testosterone. The child has XY chromosomes and testes but the childs body develops female.

Altogether there are five major factors that must align for normal sexual development: chromosomes, anatomy, hormones and brain development for gender and for sexual ideation. Most of us are very lucky to develop with all five of these in agreement. We inherit XY, have male anatomy, produce testosterone, feel comfortable in a more-or-less masculine role, and are sexually attracted to females after puberty. Or we inherit XX chromosomes, have female anatomy, produce estrogens (there are several), feel comfortable in a more-or-less feminine role, and are sexually attracted to males.

But some children are not as fortunate, and their chromosomes, anatomy, hormones, and brain development do not align. This is not uncommon. Taken in total, some form of sexual ambiguity is more common than all cases of Down syndrome and cystic fibrosis combined.

Forget the rest of the LGB alphabet. This is not about gay rights. The only group that is targeted by this legislation is transgender children. Usually by age 6, a childs brain develops a feeling of being either masculine or feminine. It is not learned. And at this age, it has nothing to do with sexual attraction.

For most of us, our feeling of being male or female will align with our anatomy. But for transgender children, this feeling of being masculine or feminine does not align with their birth anatomy. This is biological. It is not a choice. They will not decide to be a boy one day and a girl the next. These children will usually be facing hormone treatments and a series of reconstructive surgeries. But due to widespread ignorance among outside adults, some Kansas transgender children stay at home, homeschooled because of the discomfort they feel at school by the very same attitudes demonstrated in HB2171.

Masculinity or femininity is not something you learn or can change; it develops during the last trimester before birth. Dr. Dick Swaabs research team in the Netherlands was the first to clearly locate this brain difference over a decade ago.

This is a handicap, and HB2171 reflects an ignorance, indeed a cruel attitude toward those children whose brain development for gender identity does not match their birth anatomy.

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'Bathroom Bill' is discrimination - Emporia Gazette

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Windsor doctor penalized for ‘reckless’ narcotics prescribing – Windsor Star

February 13th, 2017

Windsor Star

Windsor doctor penalized for 'reckless' narcotics prescribing
Windsor Star
She reported that Barnard was administering human growth hormone to help patients with weight loss, despite the fact its use for weight loss has been discredited and rejected by the medical community. The investigator also noted numerous examples of ...

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Windsor doctor penalized for 'reckless' narcotics prescribing - Windsor Star

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Physicians’ Education Resource Plans Most Impactful Miami … – PR Web (press release)

February 13th, 2017

"I would go so far as to say there will never be another text book, old fashioned text book printed on paper, about breast cancer in our lifetime because the rate of change is too great." - Dr. Patrick Borgen

Cranbury, NJ (PRWEB) February 13, 2017

Physicians' Education Resource (PER) will host the 34th Annual Miami Breast Conference, which has been bringing together top surgical, medical, and radiation oncologists, with the aim of fostering awareness of the state-of-the-art treatments in each therapeutic area and encouraging cross team cooperation in the clinic. The conference, will be chaired by Patrick I. Borgen, MD, chair, Department of Surgery Maimonides Medical Center Brooklyn, New York.

We are in a renaissance period in the understanding of breast cancer. The rate of change, of what we do clinically, of how we put this disease into boxes, of how we predict response, has never, ever changed at a faster rate, Dr. Borgen says. I would go so far as to say there will never be another text book, old fashioned text book printed on paper, about breast cancer in our lifetime because the rate of change is too great. By the time you got the book at Barnes and Noble and got it home, it would be out of dateSo this really makes Miami important.

Miami Breast Cancer Conference will present 90 lectures in a highly interactive survey forum, which allows the top oncologists to collaborate to improve clinical pathways and clinical algorithms. The conference will also include Medical Crossfire debates where oncologists can voice their opinions on some the fields most pressing issues. This years debates include whether field radiation therapy is ready for the main stage, and whether extended hormone blockade needed for every patient diagnosed with ER positive breast cancer.

Dr. Borgen added, Everything about Miami is geared towards our mantra of hear it Friday and use it on Monday. So, this is a clinical-based conference this is like our tumor boards back home. Virtually every one of our 90 lectures has a clinical case thumbnail with a question.

The conference also includes a massive poster session, which contains 70 posters signifying some of the top breast cancer research from 15 countries, so that research can take a more global approach to breast cancer care.

About PER Since 1995, PER has been the educational resource of choice for live and online activities focusing on oncology and hematology. PER provides high-quality, evidence-based activities featuring leading national and international faculty with a focus on practice-changing advances and standards of care in treatment and disease management. Activities also include topics on emerging strategies currently under investigation, supportive care, diagnosis and staging, prevention, screening and early detection, and practice management. With the rapid advances occurring in the field of oncology, understanding how to use molecular data to diagnose and stage patients, selecting the most appropriate candidates for novel therapeutic agents, individualizing treatment based on tumor type, and referring patients to clinical trials will continue to ensure the highest level of patient care is provided. PER serves the oncology health care community, including physicians, fellows, advanced practice nurses, nurses, physician assistants, pharmacists, and researchers. PER is part of the Cranbury, N.J.-based Michael J. Hennessy Associates, Inc. family of businesses. Learn more at http://www.gotoper.com and http://www.mjhassoc.com

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Most cases of thyroid cancer are curable – Post-Bulletin

February 13th, 2017

DEAR MAYO CLINIC: How is thyroid cancer treated? Does it always require taking out the thyroid? When is iodine treatment used, and how does that work?

Treatment for thyroid cancer usually involves removing all or part of the thyroid gland. In cases where thyroid cancer is advanced or aggressive, radioactive iodine treatment may be recommended after surgery to destroy any cancer cells that couldn't be removed during surgery. For very small papillary thyroid cancers (less than 1 centimeter in diameter and completely confined to the thyroid on ultrasound examination), it may be reasonable to avoid surgery and monitor them periodically without treatment. This is termed "surveillance" and requires annual imaging of the thyroid with high-quality ultrasound. These small thyroid cancers are low risk for progression, especially in persons over 60.

The thyroid is a butterfly-shaped gland located in the midline of your neck, about halfway between your Adam's apple and your breastbone. Your thyroid gland produces two main hormones: thyroxine, or T4, and triiodothyronine, or T3.

Thyroid hormones impact many cells within your body. They maintain the rate at which your body uses fats and carbohydrates, help control your body temperature, affect the working of your nervous system, and influence your heart rate. Your thyroid gland also produces calcitonin, a hormone that helps regulate the amount of calcium in your blood.

Thyroid cancer is not common in the U.S. When it is found, though, most cases can be cured. Surgery to remove all or most of the thyroid a procedure called a thyroidectomy is often the first step in treatment.

Thyroidectomy typically involves making an incision in the center of the neck to access the thyroid gland directly. In addition to removing the thyroid, the surgeon may remove lymph nodes near the thyroid gland if the cancer is known or suspected to be spreading outside the thyroid. Then, those lymph nodes will be checked for cancer cells. An ultrasound exam of the neck before surgery can help doctors determine if lymph node removal is necessary.

When thyroid cancer is found in its earliest stage, and the cancer is very small, it may only be necessary to remove one side, or lobe, of the thyroid, and leave the rest in place. In that situation, the thyroid still can function and produce hormones.

When the entire thyroid is removed, lifelong thyroid hormone therapy is required to replace the thyroid's natural hormones and regulate the body's metabolism. In addition to supplying the missing hormone the thyroid normally makes, this medication also suppresses the pituitary gland's production of thyroid-stimulating hormone, or TSH. That's useful, because there's a possibility that high TSH levels could foster the growth of any remaining cancer cells.

If thyroid cancer is found in its later stages, if it's a more aggressive form of cancer, or if it is cancer that has come back after earlier treatment, then radioactive iodine therapy may be recommended after the thyroid has been removed.

Radioactive iodine comes in a capsule or liquid that's swallowed. The therapy works because thyroid cells naturally absorb iodine. So when the medicine is taken up by any remaining thyroid cells or thyroid cancer, the radioactivity destroys those cells. Because the thyroid is the primary site where iodine is absorbed by the body, there's a low risk of harming other cells with this treatment. Afterward, the radioactive iodine leaves the body through urine.

If thyroid cancer is not cured with a combination of surgery and radioactive iodine therapy, then chemotherapy, external radiation therapy or other treatment may be necessary. Fortunately, surgery cures most cases of thyroid cancer, and the long-term outlook after the procedure is usually excellent. John Morris III, M.D., Endocrinology, Mayo Clinic, Rochester.

Mayo Clinic Q & A is an educational resource and doesnt replace regular medical care. Email a question to MayoClinicQ&A@mayo.edu. For more information, visit http://www.mayoclinic.org.

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How This Police Officer Is Helping Trans Patients Get Better Care – Refinery29

February 13th, 2017

Gender dysphoria is one of few identities still deemed a health issue that requires diagnosis same-sex desire was removed from the Diagnostic and Statistical Manuel (DSM) in 1973. In order to medically transition and get safe access to hormones and other support, transgender people need to come out to a doctor and are often required to attend therapy.

In 2013 England's National Health Service's (NHS) changed protocol for gender dysphoria patients, asking general practitioners to refer to a gender clinic which can offer much needed services like hormone treatment, hair removal treatments, and family support groups. Yet, many GPs still refer first to a counselor who may have little to no experience with gender identity.

Gina Denham, a transgender police officer in England, is teaming up with the NHS to change that, according to Echo. It's her hope to provide trans patients in the area the support they need during transition, and get them access to one of the 7 designated NHS gender identity clinics in England.

One of the biggest barriers and potential cost to the NHS is our GPs sending our members to have counseling when they are meant to be referred to a gender clinic," Denham told Echo. "The GP just seems to ignore your request and sends you to counseling to try and cure you."

When she started to transition in 2014, Echo reports, Denham went to about 65 counseling sessions many of which were unnecessary.

Since then, Denham has become a champion for transgender rights in her community. Educating doctors is not her first project. In early 2016 Denham started a support group called Transpire for LGBTQ people in her neighborhood, which provides people the opportunity to both socialize with others who've had similar experiences and to engage in activist work. The Transpire group has helped educate medical professionals by working with the NHS on training materials.

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How This Police Officer Is Helping Trans Patients Get Better Care - Refinery29

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VistaGen Therapeutics Reports Fiscal Third Quarter 2017 Financial … – Yahoo Finance

February 13th, 2017

SOUTH SAN FRANCISCO, CA--(Marketwired - February 13, 2017) - VistaGen Therapeutics Inc. (VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, today reported financial results for the third quarter of fiscal 2017 ended December 31, 2016.

The Company also provided a corporate update, including anticipated milestones for AV-101, its new generation, orally available CNS prodrug candidate in Phase 2 development, initially for the adjunctive treatment of major depressive disorder (MDD) in patients with an inadequate response to standard antidepressant therapies approved by the U.S. Food and Drug Administration (FDA).

"We are excited about our progress during the last quarter, with several key advances related to our MDD-focused programs for AV-101, as well as potential regenerative medicine and drug rescue applications of our cardiac stem cell technology. Following productive discussions with the FDA last quarter, our team and key advisors have been working diligently to complete the diverse regulatory and technical activities necessary to support the planned launch of our Phase 2b study of AV-101 next quarter, a study we believe has game-changing potential for the millions of patients who battle MDD every day with inadequate therapies," commented Shawn Singh, Chief Executive Officer of VistaGen. "Also, our recent sublicense agreement with BlueRock Therapeutics was an important advance in our cardiac stem cell program while we remain primarily focused on our Phase 2 programs for AV-101. With potentially catalytic milestones in the coming quarters, we believe we are poised to unlock significant value for our shareholders throughout 2017," added Mr. Singh.

Recent Corporate Highlights:

The U.S. National Institute of Mental Health (NIMH) is currently conducting and fully funding a 20 to 25-patient Phase 2a study of AV-101 as a monotherapy for treatment-resistant MDD under VistaGen's Cooperative Research and Development Agreement (CRADA) with the NIMH (Phase 2a Study). Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH and a leading clinical expert on the use of ketamine for treatment-resistant MDD, is the Principal Investigator of the Phase 2a Study. Following recent guidance from the NIMH, the Company currently anticipates that the NIMH will complete the Phase 2a Study by the end of 2017.

VistaGen is preparing to launch a 280-patient, multi-center, double-blind, placebo controlled Phase 2b efficacy and safety study evaluating AV-101 as a new generation adjunctive treatment for MDD patients with an inadequate response to standard, FDA-approved antidepressant therapies. The Company currently anticipates commencing patient enrollment in the Phase 2b Study in the second quarter of 2017. Dr. Maurizio Fava of Harvard University Medical School will serve as the Principal Investigator of VistaGen's AV-101 Phase 2b Study. Topline clinical results from the Phase 2b Study are currently anticipated by the end of 2018.

Dr. Mark Smith, Chief Medical Officer of VistaGen, commented, "We look forward to starting patient enrollment in our Phase 2b study of AV-101 as an adjunctive therapy in the treatment of MDD. We believe we have significantly de-risked this Phase 2b study with a clinical trial methodology that is designed to overcome the challenge of placebo effects in psychiatric clinical trials. Based on the study protocol we have designed in collaboration with key opinion leaders in depression and neuroscience, including our Principal Investigator, Dr. Fava, we expect that achieving a successful outcome of our Phase 2b study will be integral in realizing AV-101's potential to displace atypical antipsychotics and non-drug interventions in the current depression treatment paradigm, representing a much needed treatment solution for physicians and patients, as well as an enormous opportunity for VistaGen."

Expected Near-Term Milestones:

"The NIMH recently updated us on their timelines for the completion of the Phase 2a study of AV-101 as a monotherapy for MDD. The Phase 2a study protocol requires considerable time and dedication from both the study participants and the multi-disciplinary NIMH teams involved. Patient enrollment for the Phase 2a study remains ongoing and we currently anticipate the NIMH's completion of the study by the end of 2017. Our top priority is to execute our plans for our Phase 2b study of AV-101 as a new generation adjunctive treatment of MDD, and we remain on track to launch that important study in the second quarter. As part of our Phase 2 program, this Phase 2b study has been specifically designed to achieve important outcomes that will be key to advancing AV-101 into a pivotal program in MDD and more broadly beyond MDD, as we continue to advance our global commercialization strategy. We are confident that our Phase 2 program is a major step forward in positioning AV-101 as a potentially transformative adjunctive treatment of MDD and other CNS disorders," concluded Mr. Singh.

Stem cell registry drive at SAU seeks to connect potential donors with people who need help – Magnoliareporter

February 13th, 2017

When three Southern Arkansas University nursing students started organizing this weeks stem cell registry drive more than three months ago, they were not aware that a member of the Mulerider family is one of more than 1,400 whose life could be saved.

The stem cell/bone marrow registry drive is scheduled for 9 a.m.-3:30 p.m. on Tuesday and Wednesday both in the Reynolds Center Rotunda and the SAU Baptist Collegiate Ministry.

For more information, contact Dr. Becky Parnell at (870)235-4365 or at bbparnell@saumag.edu .

The SAU BSN students initially behind the project are Renee Langley, Tabitha Elliott and Courtney Owens. Parnell explained that while attending the Arkansas Student Nurses Association annual meeting in Little Rock, the students were introduced to the need for bone marrow donors. They even registered to be possible donors themselves.

Parnell said they realized this project was a perfect example of how nurses can impact the care of people outside the normal hospitalized patient.

They recognized how many people this could potentially impact and wanted to recruit more people (to register), said Parnell. I have seen the bone marrow process it is truly a life-saving intervention for many people that are devastated by leukemia.

When Parnell began promoting the registry event on campus, it was brought to her attention that Sydney Galway, the daughter of a Magnolia native, 1984 SAU alum and Board of Governors Chair Beth Galway, is suffering with acute myeloid leukemia.

Sydney Galway is in dire need of a bone marrow transplant.

When Sydney was diagnosed with acute myeloid leukemia, the doctors told us that Sydneys only cure would come from a bone marrow transplant. The doctors were, and are, confident of the success of her treatment due to the fact that she has a high chance to find a perfect bone marrow donor, said Galway.

Her increased chance of finding a match, Galway explained, is simply because she is a Caucasian female which has one of the highest bone marrow donor rates. She has a 97 percent chance to find a donor.

Of course, the first donor they looked at was her sister. A sibling has only a 25 percent chance to be a match; a parent even less. Sydneys sister was not a match, said Galway.

Donor matches are generally based on race. With todays diverse community, the need for bone marrow donors from minority and mixed race groups is high. An African American patient has only a 66 percent chance to find a match.

The doctors and nurses that I have talked to indicate that the need is huge for African Americans as well as donors from India, said Galway.

She said that the treatment for Sydney, who is a sophomore in college, is now in phase 3. Her next step is a bone marrow transplant.

We hope to have a perfect match for her and pray that the donor will be willing to do all that is necessary for providing the blood or bone marrow needed for the transplant, said Galway.

The drive is being sponsored by SAUs Department of Nursing and University Health Services. Junior and senior BSN students will also be assisting in the bone marrow drive as a professional development activity.

Becoming a member of a stem cell/bone marrow registry only requires that you provide a swab of the cells inside your cheek. To register is a painless and fast way to possibly save a life.

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Stem cell registry drive at SAU seeks to connect potential donors with people who need help - Magnoliareporter

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SNA strives to find bone marrow donors at MU – MU The Parthenon

February 13th, 2017

Saving a life can begin with a simple swab of a cheek.

Marshalls Student Nurses Association is trying to help accomplish this goal through a bone marrow registry drive with Be The Match, a nonprofit organization, Feb. 15 in the Memorial Student Center. Anyone from ages 18 to 44 with no major preexisting diseases are eligible to register.

The main idea is that Be The Match connects critically ill patients with a life-saving bone marrow donor, senior nursing major Molly Arthur said. Most patients do not find a marrow match within their own family, so they have to rely on a complete stranger to donate to them.

The SNA decided to do this drive after meeting several patients through their clinicals at Cabell Huntington Hospital who have the possibility of receiving a bone marrow transplant through the course of their treatment.

I know a little boy who went recently to see if he had any matches to get a transplant, and they had 10 people that were matches for him, senior nursing major Jenna Fields said. If he would need one later on, they would wipe out his immune system and replace it through the bone marrow to fight off the disease.

In order to register, donors will go through a series of questions about their medical history and will have their cheeks swabbed to collect cells, which will take about 10 minutes. According to Be The Match registry, only one in every 430 people go on to donate.

There are three ways to donate: peripheral blood stem cells through an IV, bone marrow through the hip by a surgical procedure and cord blood after giving birth.

They put an IV in, they take the blood out and spin out what they need and everything else goes back into your body. Its just like giving blood, and you potentially save a life, senior nursing major Alison Evans said.

The registry drive is taking place in the Don Morris Room from 11 a.m. to 5 p.m. Jan. 15. The SNA has a goal of registering 100 donors.

The more people on the registry, the more likely you are to find a match, Evans said. The goal is to get as many people on the registry as possible to potentially raise someones percentage of finding a match.

Heather Barker can be contacted at [emailprotected]

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Lion-hearted fighter beats the odds – The Straits Times

February 13th, 2017

Ten-year-old Boon Kye Feng prances around the living room in furry purple pants that match the lion's head he is wearing.

He lifts the head and moves it from side to side to a beat only he can hear.

Even when the little lion gets thirsty, he drinks water through the opening in the head.

Seeing him at play, it may be difficult for strangers to tell that he has spent almost half his life battling leukaemia.

His family fought it along with him, gifting two transplants - cord blood from his baby sister and stem cells from his mother - to keep him alive.

MIRACLE BOY

I believe Kye Feng is a 'miracle'. We have all learnt a lot from him, not only in the science of managing the disease and the doctor-patient relationship, but also in his love of life, and his fearlessness and resilience, despite the years of pain and suffering.

ASSOCIATE PROFESSOR TAN POH LIN, from the paediatric haematology- oncology division of NUH.

Despite the intensive treatment, his parents said he had remained positive and playful.

It had started in late 2011 when Kye Feng developed spots and bruises which his parents thought were sandfly bites.

When the spots appeared a second time, his mother, Mrs Celine Boon, decided to take him for a check-up.

Doctors found that his white blood cell count was very high and told the family he could have leukaemia (cancer of the blood).

It was diagnosed as juvenile myelomonocytic leukaemia (JMML), a rare form of the disease.

But Mrs Boon, 38, was not too surprised.

This was because Kye Feng and his twin brother, Kye Teck, had previously developed juvenile xanthogranuloma (JXG), a skin disorder that is usually benign and self-limiting.

They also have an older sister, now 16, who was unaffected.

While reading up on JXG earlier, Mrs Boon had come across a potential link to JMML.

She said: "Still, I had never expected that it would happen to my son. I was quite alarmed."

JMML is so rare that blood samples had to be sent to Germany to confirm the diagnosis.

Kye Feng began chemotherapy at KK Women's and Children's Hospital (KKH) in 2012 to control the condition while waiting for a bone marrow transplant.

Although KKH doctors had not seen a JMML case in about 10 years, they did the transplant as there were few other options.

His father, Mr Roy Boon, 46, said: "It was all trial and error. There's no exact treatment for JMML."

Mrs Boon was then pregnant with their fourth child and doctors said the baby girl's cord blood could be used for the transplant as there is a 25 per cent chance of a match between siblings.

Juvenile myelomonocytic leukaemia (JMML) is a very rare form of childhood leukaemia. The hallmark symptom of the disease is the increased number of white blood cells known as monocytes.

Normal monocytes protect the body from infections, but those in patients with this leukaemia are cancerous and reproduce uncontrollably. The monocytes may then infiltrate organs such as the liver, spleen, lungs, lymph nodes and even skin.

In Western countries, one in a million children are afflicted with the disease each year. Based on Singapore population statistics last year, there is an average of one case every three years.

For the majority of JMML patients, a haematopoietic - or blood forming - stem cell transplant (HSCT) is the only curative option.

Stem cells are cells that have the potential for self-renewal and differentiation. They can develop into different forms, including white blood cells, red blood cells and platelets. Such a transplant can help patients develop new and healthy blood cells.

Stem cells can be found in the bone marrow, blood, fat tissue and placenta. They are abundant in the bone marrow but, even so, make up only 1 per cent of all cells there.

They can be "harvested" directly from the bone marrow or from the blood, whether they are from an adult volunteer or from umbilical cord blood.

The bone marrow must be stimulated to coax or force the stem cells into the peripheral blood system, but techniques are well-tested and safe.

After undergoing HSCT, 50 per cent of the patients will go on to become long-term survivors.

Abigail Ng

Source: Associate Professor Tan Poh Lin, senior consultant at the division of paediatric haematology-oncology, National University Hospital.

Thankfully, it was a full match for Kye Feng, who had the transplant and recovered well.

He looked forward to starting Primary 1 with his brother.

But before the March holidays of his first year in school, doctors noticed that the percentage of donor cells in him was beginning to fall, signalling that there could be a problem.

When it became clear that the cancer had returned, Mrs Boon said she broke down and cried.

"I was shocked. There weren't any physical symptoms. Why did it happen so quickly? It wasn't even one year after the transplant and things had looked so promising," she said.

A SECOND CHANCE

The family sought a second opinion from the National University Hospital (NUH) and entered into the care of Associate Professor Tan Poh Lin from the paediatric haematology-oncology division.

While doctors from both hospitals suggested a second transplant for Kye Feng, there was more bad news.

His illness was mutating into mixed-phenotype acute leukaemia, a combination of two forms of cancer.

He also faced a life-threatening infection that caused high fever and bloating.

Besides beginning palliative care to improve his quality of life, the family continued to push for treatment, including natural killer-cell therapy and the removal of Kye Feng's enlarged spleen in a complicated seven-hour operation.

Even though the test results showed that leukaemic cells remained in his bone marrow, Kye Feng had a second transplant in September 2015, this time using stem cells from his mother.

Doctors usually recommend transplants only when patients register no leukaemic cells.

Mrs Boon said: "If he didn't have the transplant, he would have only six months more. With the transplant, he would at least have a chance of recovery.

"He was fighting hard. If I didn't give him the chance, I would never know if he could have survived."

Kye Feng responded well to his mother's stem cells.

Dr Tan said: "I believe Kye Feng is a 'miracle'. We have all learnt a lot from him, not only in the science of managing the disease and the doctor-patient relationship, but also in his love of life, and his fearlessness and resilience, despite the years of pain and suffering."

The crucial three months after the transplant passed by without issue, but the boy developed a graft versus host disease (GVHD) one year later.

Still, his parents were relieved that it was not a second relapse.

He was put on medication for GVHD and will recover completely.

In the meantime, the family is treasuring the time they can spend together.

Mrs Boon said: "We will relax and go with the flow, as long as Kye Feng is happy."

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Lion-hearted fighter beats the odds - The Straits Times

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Scientists discover an unexpected influence on dividing stem cells’ fate – ScienceBlog.com (blog)

February 13th, 2017

When most cells divide, they simply make more of themselves. But stem cells, which are responsible for repairing or makingnew tissue, have a choice: They can generate more stem cells or differentiate into skin cells, liver cells, or virtually any of the bodys specialized cell types.

As reported February 3 in Science, scientists at The Rockefeller University have discovered that this pivotal decision can hinge on whether or not tiny organ-like structures, organelles, are divvied up properly within the dividing stem cell.

In order for the bodys tissues to develop properly and maintain themselves, renewal and differentiation must be carefully balanced, says senior author Elaine Fuchs, the Rebecca C. Lancefield Professor and head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Development. Our experiments suggest an unexpected role for the positioning and inheritance of cellular organelles, in this case enzyme-filled peroxisomes, in controlling this intricate balance.

An uneven division

The outer section of the skin, the epidermis, provides a protective barrier for the body, and stem cells reside deep within it. During development, these cells divide so that one renewing stem cell daughter remains inward while the other daughter differentiates and moves outward to become part of the epidermis outer layers. First author Amma Asare, a graduate student in the lab, wanted to know how skin cells first emerge and begin this transition.

Looking in developing mouse skin, Asare devised an approach to identify genes that help guide the balance between new cells that either stay stem-like or differentiate. One particular protein, Pex11b, caught her attention. It is associated with the membrane that surrounds the peroxisome, an organelle that helps to free energy from food.

Asare showed that the protein seems to work by making sure the organelles are in the right locations so they can be divided between the daughter cells. In cells that lacked Pex11b, peroxisomes werent divvied up evenlyin some cases, one daughter cell ended up with all of the peroxisomes and the other didnt get any at all. And for those cells whose peroxisome distribution was disrupted, cell division took longer, and the mitotic spindle, the structure that separates the daughters genetic material, didnt align correctly.

The net result of depleting skin stem cells of Pex11b, Asare found, was that fewer daughter cells were able to differentiate into mature skin cells.

A delay changes fate

The researchers next moved peroxisomes around in the cell using a sophisticated laboratory technique, and the effect was the same. If the peroxisomes are in the wrong positions during cell division, no matter how they get there, that slows down the process, Asare says.

The effect for the whole organism was dramatic: If peroxisome positioning was disrupted in the stem cells, the mice embryos could no longer form normal skin.

While some evidence already suggested the distribution of organelles, including energy-producing mitochondria, can influence the outcome of cell division, we have shown for the first time that this phenomenon is essential to the proper behavior of stem cells and formation of the tissue, says Fuchs, who is also a Howard Hughes Medical Institute Investigator.

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Study implicates neural stem cell defects in smooth brain syndrome – Medical Xpress

February 13th, 2017

February 13, 2017 by Nicholas Weiler

Research led by scientists at UC San Francisco and Case Western Reserve University School of Medicine has used brain "organoids"tiny 3-D models of human organs that scientists grow in a dish to study diseaseto identify root causes of Miller-Dieker Syndrome (MDS), a rare genetic disorder that causes fatal brain malformations.

MDS is caused by a deletion of a section of human chromosome 17 containing genes important for neural development. The result is a brain whose outer layer, the neocortex, which is normally folded and furrowed to fit more brain into a limited skull, instead has a smooth appearance (lissencephaly) and is often smaller than normal (microcephaly). The disease is accompanied by severe seizures and intellectual disabilities, and few infants born with MDS survive beyond childhood.

In the new studypublished online January 19, 2017 in Cell Stem Cellthe research team transformed skin cells from MDS patients and normal adults into induced pluripotent stem cells (IPSCs) and then into neural stem cells, which they placed in a 3 dimensional culture system to grow organoid models of the human neocortex with and without the genetic defect that causes MDS.

Closely observing the development of these MDS organoids over time revealed that many neural stem cells die off at early stages of development, and others exhibit defects in cell movement and cell division. These findings could help explain how the genetics of MDS leads to lissencephaly, the authors say, while also offering valuable insights into normal brain development.

"The development of cortical organoid models is a breakthrough in researchers' ability to study how human brain development can go awry, especially diseases such as MDS," said Tony Wynshaw-Boris, MD, PhD, chair of the Department of Genetics and Genome Studies at Case Western Reserve University School of Medicine, and co-senior author of the new study. "This has allowed us to identify novel cellular factors that contribute to Miller-Dieker syndrome, which has not been modeled before."

'Smooth Brain' Organoids Reveal Defects

Previous research on the causes of lissencephaly has relied on mouse models of the disease, which suggested that the main driver of the disorder was a defect in the ability of young neurons to migrate to the correct location in the brain. But Arnold Kriegstein, MD, PhD, professor of neurology, director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF, and co-senior author, says there are significant drawbacks to this approach.

"Unlike the human brain, the mouse brain is naturally smooth," Kriegstein said. "If you are studying a disease that leads to a smooth brain in humans, it's a challenge to study it in an animal that normally has a smooth brain."

The mouse brain also lacks a type of neural stem cell called outer radial glia, which were discovered by Kriegstein's group in 2010. These cells are thought to have played a crucial role in the massive expansion in size and complexity of the primate brain relative to other mammals over the course of evolution.

In order to more accurately model the progression of MDS in the embryonic human brain, study first author Marina Bershteyn, PhD, a postdoctoral researcher in the Wynshaw-Boris and Kriegstein labs, spearheaded the development of MDS cortical organoids and techniques to observe how stem cells within these organoids developed in the laboratory into the different cell types seen in first-trimester embryonic human brains.

Bershteyn and her team found using time-lapse imaging that outer radial glia cells that grew in patient-derived organoids had a defect in their ability to dividepotentially contributing to the small, smooth brains seen in MDS patients.

"There are just fundamental differences in how mouse and human brains grow and develop," said Bershteyn, who is now a scientist at Neurona Therapeutics, a company founded by Kriegstein and colleagues to develop stem cell therapies for neurological diseases. "Part of the explanation for why these observations were not made before is that outer radial glia cells are quite rare in mouse."

In addition, the team found that early neural stem cells called neuroepithelial cells which are present in both mice and humans die at surprisingly high rates in MDS organoids, and when they do survive, divide in an abnormal wayas if they are prematurely transforming into neurons, cutting short important early stages of brain development.

Consistent with prior mouse studies, time-lapse imaging also revealed that newborn neurons are unable to migrate properly through developing brain tissue, which potentially contributes to the failure of MDS brains to properly form outer brain structures.

Organoid Research Opens Doors to Studying Human Brain Diseases in Lab

Together, these observations helped the team pinpoint developmental stages and specific neural cell types that are impaired in MDS. The next step to understanding lissencephaly more broadly, the authors say, will be to test cells from patients with different genetic forms of the disease, so researchers can begin to link specific mutations with the cellular defects that drive brain malformation.

The study is also a demonstration of the utility of patient-derived brain organoids as a way to bridge the gap between animal models and human disease, the authors say. In particular, the finding that human outer radial glia cells readily grow in organoid models opens the door for scientists worldwide to study the role of these cells in both normal human brain development and disease.

"Patient-derived cortical organoids are creating a huge amount of excitement," Kriegstein said. "We are now able to study human brain development experimentally in the lab in ways that were not possible before."

Explore further: Scientists engineer gene pathway to grow brain organoids with surface folding

More information: Marina Bershteyn et al. Human iPSC-Derived Cerebral Organoids Model Cellular Features of Lissencephaly and Reveal Prolonged Mitosis of Outer Radial Glia, Cell Stem Cell (2017). DOI: 10.1016/j.stem.2016.12.007

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CRISPR’s breakthrough problem | February 13, 2017 Issue – Vol. 95 … – The Biological SCENE

February 13th, 2017

In brief

In fewer than five years, a gene-editing technology known as CRISPR has revolutionized research. Now, many are wondering if it can do the same for medicine. Several companies are hoping to commercialize CRISPR-based therapies that could potentially offer a permanent fix for a vast array of genetic diseases. But theres a catch: Getting CRISPR into the body, across cell membranes, and into human DNA is no simple feat. Read on to learn how chemists and bioengineers are joining the CRISPR craze to solve gene editings delivery dilemma.

In fewer than five years, an important new gene-editing tool called CRISPR has radically changed the face and pace of biological research. The ability to quickly and cleanly remove and replace stretches of DNA has already inspired thousands of publications featuring the technique and led to the creation of a slew of biotech businesses hoping to capitalize on CRISPR.

CRISPRs power to effortlessly target and tweak any piece of DNA seems limitless. Thomas Barnes is the chief scientific officer of the CRISPR-centered Intellia Therapeutics, whose founders include one of the inventors of CRISPR, Jennifer Doudna. He says there is an ever-growing backlog of well-understood rare genetic conditions with little that people can do about them. Barnes hopes CRISPR will change that.

By tackling genetic disease at its rootsmutations in the DNACRISPR could end thousands of ailments, Barnes and others believe. Multiple research groups and companies are hot on the tracks of unleashing CRISPR on sickle cell disease, hemophilia, cystic fibrosis, Duchenne muscular dystrophy, genetic forms of blindness, and, of course, cancer.

The hype is partly about CRISPRs broad applicability, but CRISPRs true promise is its potential for a one-and-done cure. Changing your DNA is a permanent fix. CRISPRshort for the clustered regularly interspaced short palindromic repeats in the bacterial immune system from which the technology was derivedis a two-part system: a customizable guide RNA and a protein called Cas9. The guide RNA directs Cas9 to any desired segment of DNA for editing. The Cas9 enzyme then cuts the DNA at that precise location, allowing for genes to be turned on or off or for the removal or insertion of DNA.

But editing the DNA of cells in a petri dishor even curing a mouse of a diseaseis one thing; making the hot new technology work in humans is a whole other challenge. Sneaking the gene-editing complex into human cells is no easy task.

It will take some fancy molecular maneuvering to get the bulky Cas9 protein and the negatively charged guide RNA into humans. To work its magic, the unwieldy gene-editing system first needs to get into the body, skirt past the immune system, and infiltrate its target tissue. From there, it must sneak across cell membranes, escape the acidic environment of the cells endosomes to find the nucleus, and then home in on the correct location on the DNA. In other words, CRISPR has a drug delivery problem.

The Cas9 enzyme and the guide RNA composing the CRISPR complex cannot be swallowed in pill form or simply injected into the bloodstream. And a one-size-fits-all package is unlikely to work for every condition, so researchers are eagerly testing old strategies and creating new ones to achieve a CRISPR cure.

David Liu of Harvard University says this delivery dilemma isnt unusual for a new gene-editing technology, but researchers now feel this incredible urgency and excitement because of the promise of using CRISPR for therapeutic applications. Since its inception as a gene-editing tool in 2012, nearly 5,000 papers mentioning CRISPR have been published in PubMed. The CRISPR craze is reeling in polymer chemists, drug delivery designers, and bioengineers all helping move CRISPR from the lab bench to the doctors office.

Ive just never seen any field that progresses at this pace, says Niren Murthy of the University of California, Berkeley, who cofounded a start-up called GenEdit, dedicated to CRISPR delivery, in February 2016. There is nothing comparable to the competitiveness of the CRISPR field, he says.

From a delivery perspective, I am sure there will be all sorts of surprises, says Kathryn Whitehead of Carnegie Mellon University. C&EN spoke with more than 30 academic and industry researchers about CRISPRs delivery dilemma. Some expect success soon. Others are trying to temper expectations, pointing to the historically long time horizon for turning new technologies into treatments. But as Whitehead says, If this is possible, everything changes.

CRISPR gene editing is derived from a primordial immune system in bacteria called clustered regularly interspaced short palindromic repeats. A guide RNA, which is complementary to a target DNA sequence, directs the Cas9 enzyme (light blue) to a specified location for DNA cutting. Some applications require an additional DNA template (not shown) to fill in the cut. Source: Adapted from OriGene Technologies

CRISPR isnt the first gene-editing technology promising to cure thousands of diseases. In fact, multiple studies of treatments developed using older technologies are now under way. Drug delivery guru Daniel Anderson of Massachusetts Institute of Technology points out that one of the most advanced programs is Sangamo Biosciences ongoing clinical trial to remove T cells from patients, edit their DNA to make them resistant to HIV, and reinject the modified cells. So presumably, there are some genome-edited people walking around in California that they helped create, Anderson says.

Sangamo is using an older gene-editing tool called zinc finger nucleases, a complex protein structure designed to bind and cleave a specific region of DNA. And doctors at the Great Ormond Street Hospital in London recently reported using a similar gene-editing technique called TALENs, which also recognizes and cuts precise DNA sequences, to engineer immune cells for a therapy that may have cured two infants of leukemia.

Both technologies have been around for longer than CRISPR has, with zinc-finger-based editing being in the works for more than two decades. They also both suffer from a limitation that has inhibited their widespread adoption: Each is a cumbersome protein complex that needs to be individually engineered for every new DNA target.

CRISPR, meanwhile, is easily adaptable. The Cas9 cutting protein remains the same for all applications, and to make a new edit, researchers need only to switch out the guide RNA. If the DNA sequence that needs editing is known, securing the complementary guide RNA is as easy as clicking Order from a supplier.

When CRISPR came along, everyone knew what to do with it, Intellias Barnes says. People had been going around in a go-kart and you gave them a Ferrari, so away they go.

Jacob Corn of UC Berkeley says cheap and easily customizable guide RNA empowers the democratization of gene editing. Corns lab is one of several using CRISPR to cureat least in isolated cells and micesickle cell disease, where a single-letter DNA mutation stymies the oxygen-ferrying capacity of red blood cells.

Corn envisions a world where patient DNA testing is coupled to CRISPRs customizability, and scientists can easily whip up a fix for problematic genetic mutations. I think that, in the future, well be able to tackle genetic diseases with the same speed we can diagnose them, he says.

Corns dream might not be far off, at least for blood disorders such as sickle cell disease. In that condition, stem cells collected from the blood or bone marrow could be removed from a patient, edited in the lab to correct the DNA typoa process called ex vivo gene editingand then reinjected to proliferate and make a patient healthy.

Editing cells harvested from a patient is relatively straightforward. Researchers commonly use electroporation, a technique that uses an electric pulse to momentarily create pores that allow the Cas9 protein and guide RNA complex to slip inside cells in a dish. This technique has the potential to address hematological disorders and is also being used to beef up immune cells to fight cancers such as leukemia.

Lloyd Klickstein, head of translational medicine for the new indications discovery unit at the Novartis Institutes for BioMedical Research, says, Thus far, the ex vivo technologies are whats been done, and thats what most of the companies are looking to do first, Novartis included.

The concept looks promising on paper, but no one knows how well it will work in humans. Chinese researchers at Sichuan University claimed to be the first to do ex vivo therapy with a handful of people with cancer last year, and University of Pennsylvania researchers are gearing up for a similar clinical trial in Philadelphia, San Francisco, and Houston this year.

Although researchers are excited about the potential to use CRISPR to create therapies from peoples own blood, immune, and stem cells, thousands more genetic conditions affect everything else. For those disorders, CRISPR needs to be delivered like more traditional medicines so it can work its wonders editing DNA inside the body. But the challenge of shuttling CRISPR directly to the diseased tissue, or in vivo gene editing, is so daunting it could stall CRISPRs otherwise rapid advancement.

The first in vivo CRISPR therapy to be tested in humans will likely borrow its delivery vehicle from the world of gene therapy, where hollowed shells of viruses are used to transport genes inside cells and then, in theory, permanently produce a therapeutic protein. Decades of gene therapy research has yielded a reasonably good carrier for genetic material, the adeno-associated virus (AAV). Compared with other viral vehicles, the immune system tends to ignore AAV, and the carrier is able to target specific cell types in the body.

Editas Medicines founding scientific adviser is one of CRISPRs inventors, Feng Zhang of the Broad Institute. Editas is using AAV to deliver CRISPR in monkeys. In this study, CRISPR targets the genetic mutation that causes Leber congenital amaurosis 10, a rare form of progressive childhood blindness. The biotech firm plans to ask FDA for permission to start human studies of the treatment, which must be directly injected into the eye, by the end of this year. We chose that disease because we felt that we could deliver our machinery there, says Charles Albright, chief scientific officer of Editas.

Seokjoong Kim, research director at the South Korea-based gene-editing company ToolGen, is also conducting CRISPR experiments in mice for eye disorders, including age-related macular degeneration and diabetic retinopathy. ToolGen will also deliver CRISPR with AAV because it is the most validated delivery tool clinically, Kim says. He notes that AAV is already being used in gene therapy clinical trials for Parkinsons disease, hemophilia, and vision disorders.

We are building on decades of work in gene therapy, Albright says. We believe that patients and regulators and physicians will feel more comfortable using this method. Using CRISPR in humans is enough of an unknown for Editas and ToolGen, and they believe the chances of success and drug approval are higher with an established delivery system such as AAV.

But AAVs strength for gene therapyperpetual production of a proteinis its drawback for gene editing. One of the potential issues with AAV is that there is no good way to control the expression of Cas9, says Mark Kay, director of the Program in Human Gene Therapy at Stanford University. Once inside cells, the DNA plasmid will continue producing the Cas9 enzyme indefinitely. Since CRISPR needs to make its edit only once, the longer Cas9 hangs out inside the cell, the greater the chance the enzyme will make unwanted cuts in a patients DNA, Kay says.

Those off-target cuts are frequent topics of concern among CRISPR scientists. Even though the tool is precise, there is no guarantee it will perform to perfection. That liability has driven many researchers to look for other ways of delivering CRISPR.

The challenge of commercializing CRISPR has an even closer cousin than gene therapy. Work by scientists in 1998 unexpectedly showed that double-stranded RNA molecules could suppress the translation of messenger RNA (mRNA) into protein. Known as RNA interference, or RNAi, the research garnered a Nobel Prize in 2006 and spurred the creation of start-ups aimed at turning this powerful method of silencing genes into therapies.

But RNA cannot be directly injected into the bloodstream, where it gets degraded and triggers an immune reaction. So scientists have spent the past decade figuring out how to get their molecules inside cells. Now, CRISPR researchers are hoping to borrow their most common delivery vehicle, the lipid nanoparticle.

To reuse lipid nanoparticles for CRISPR, the gene-editing system has to be packaged in a way that recapitulates the negatively charged RNA molecules used in RNAi. Instead of delivering Cas9 as a functional protein, many researchers are sticking the mRNA instructions to make Cas9 inside their nanoparticles and letting the cell produce the protein.

Andersons lab at MIT has been a center of thought for this research. Hao Yin, a postdoctoral researcher in Andersons lab, packaged Cas9 mRNA in lipid nanoparticles previously developed in the Anderson lab for shipping RNAi molecules across a cells lipid membrane. Yin then delivered that alongside guide RNAs packaged separately in AAV to fix broken genes in mice with liver disease. The upside of the method is minimal off-target cutting by Cas9. The downside is that the efficiency is very low, Yin says. Only about 6% of hepatocytes, or liver cells, were edited by CRISPR (Nat. Biotechnol. 2016, DOI: 10.1038/nbt.3471).

New and improved lipid nanoparticles are popping up that can deliver both Cas9 mRNA and the guide RNA in the same particle. Although that dual packaging would in theory improve editing efficiency, it also poses some logistical problems. The molecules used in RNAi are only about 20 nucleotides long. Guide RNAs used in CRISPR, on the other hand, are about 100 nucleotides long, and the mRNA encoding Cas9 is an unwieldy beast of 4,500 nucleotides. So if you take the off-the-shelf lipid nanoparticle formulation and instead encapsulate the CRISPR system, its just not very good, says Daniel J. Siegwart of the University of Texas Southwestern Medical Center.

Siegwart, who was previously a postdoctoral researcher in Andersons lab, became the first to successfully deliver that kind of dual packaging to mice in December. Lipid nanoparticles require several ingredients for ferrying RNA into cells, including positively charged lipids for binding the negatively charged RNA. Siegwarts group synthesized zwitterionic amino lipids, ones containing both positive and negative charges, which help bind, stabilize, and release the mRNA as the particles cross into cells (Angew. Chem. Int. Ed. 2016, DOI: 10.1002/anie.201610209).

Lipid nanoparticles enter cells through a pinched-off cell membrane envelope called the endosome, and getting Cas9 mRNA out of that structure can be another limiting step. In January, Paul A. Wender and Robert M. Waymouth of Stanford University unveiled a polymer nanoparticle system to overcome this problem. Their particle acts like a physical property chameleon, Wender says, changing its form as it crosses the cell membrane and enters the endosome.

Wender and Waymouths system, called charge-altering releasable transporters, are made of initially positively charged oligo(-amino ester) polymers that bind the negatively charged mRNA. Upon entering the endosome, where the pH becomes more acidic, positively charged amine molecules in the polymer become neutrally charged amides, which releases the mRNA into the cell (Proc. Natl. Acad. Sci. USA 2017, DOI: 10.1073/pnas.1614193114). Although their paper didnt explicitly test the concept on Cas9 mRNA, thats on the to-do list.

Lipid nanoparticle innovation may be blossoming, and CRISPR developers are confident they can reach the clinic more quickly and safely than with RNAi, but that delivery vessel is by no means foolproof.

Rodger Novak, chief executive officer of CRISPR Therapeutics, whose founders include another of CRISPRs co-inventors, Emmanuelle Charpentier, points out that CRISPR has an advantage over RNAi, which turns down protein production only temporarily and needs to be readministered periodically. Those repeat injections can cause liver toxicity, a side effect that has slowed down the initially rapid progress of RNAi companies. Although the technology is maturing, there are no approved RNAi drugs.

The whole lipid nanoparticle field is a little bit weird, Ross Wilson of UC Berkeley says. The literature is full of success stories that are never followed up on; they just fizzle.

Wilson is one of several researchers working on delivering Cas9 as a protein rather than as mRNA in a lipid nanoparticle or as DNA in a virus. Researchers call this form of CRISPR a ribonucleoprotein, which is the active form of the guide RNA hooked up to the Cas9 enzyme in a single, ready-to-go complex.

David Liu of Harvard University says delivering CRISPR in a virus gives the least amount of control because it manufactures the Cas9 protein indefinitely. If there are too many Cas9 enzymes in a cell, there is a greater chance that one of them may accidentally cut DNA in the wrong place. Directly delivering the protein gives the most control because lower levels of Cas9 in each cell means a lower risk of potentially dangerous off-target cutting, Liu says. His group developed cationic lipid nanoparticles for CRISPR ribonucleoprotein delivery (Nat. Biotechnol. 2015, DOI: 10.1038/nbt.3081).

Liu, along with Qiaobing Xu of Tufts University, also created lipid nanoparticles that are biodegradable inside cells. The system binds negatively charged CRISPR ribonucleoproteins initially, but releases them upon entering the chemically reducing environment of the cell (Proc. Natl. Acad. Sci. USA 2016, DOI: 10.1073/pnas.1520244113).

Wilson is looking to find a way to deliver CRISPR ribonucleoproteins without the hassle of lipid nanoparticles. To do that, he needs to make the ribonucleoprotein complex stable in the bloodstream, able to escape the cells endosome, and even able to home in on a particular tissue type. But there is a downside. The immunogenicity of Cas9 could be a real issue, Wilson says.

Other scientists are crafting even more exotic delivery systems for CRISPR, including a yarn ball-like structure called a DNA nanoclew developed by Chase Beisel and Zhen Gu of North Carolina State University. Their nanoclew uses repeated stretches of DNA complementary to the guide RNA wrapped up in a ball to deliver Cas9 protein to cells. (Angew. Chem. Int. Ed. 2015, DOI: 10.1002/anie.201506030).

Even as the field works out the delivery kinks, therapies are expected to soon reach people. Clinical trials using ex vivo gene editing in humans with CRISPR is anticipated to start in the U.S. this year, with in vivo gene editing likely in 2018 and 2019.

Casebia Therapeutics, a joint venture between Novartis and CRISPR Therapeutics, is making its commitment to the delivery challenge clear, with plans to hire a head of delivery. James Burns, CEO and president of Casebia, says, There are some approaches that we can take now, but to really harness or achieve CRISPRs full potential, we are going to have to invest in new delivery technologies. Currently, most CRISPR-based companies are taking an agnostic, whatever works approach, testing both AAV and lipid nanoparticles for their first rounds of treatment.

Berkeleys Corn points out another problem with CRISPR that many people conveniently gloss over. We are really good at breaking sequences and not really good at fixing them, he says. Some conditions can be cured using Cas9 to cut out a mutation or turn a gene off. But there are many more conditions where faulty DNA needs actual correcting. That requires a third component: a DNA template strand to tell the cells repair machinery how to fill in a cut made by Cas9.

Delivering all three has been really challenging and it has not been demonstrated in in vivo systems with any lipid nanoparticles yet, says Kunwoo Lee, who is now CEO of the start-up GenEdit that he founded in February 2016 shortly before finishing his Ph.D. in Murthys lab at Berkeley. GenEdit focuses on applications of CRISPR that will require a DNA template for repair.

Lee and his colleagues at GenEdit already have a few scientific studies under review, including one that uses gold nanoparticles as a core material to load the three components of the CRISPR system. They are also working on lipid and polymer nanoparticle systems, all designed to deliver CRISPR ribonucleoproteins. Although that strategy is promising for minimizing off-target cutting, it may also be the furthest away from being an injectable treatment in the clinic.

There is simply no way that any particular delivery modality is going to provide the means to address all of those targets, so it really needs to be an all-of-the-above approach, says Erik Sontheimer of the RNA Therapeutics Institute at the University of Massachusetts Medical School. And from the looks of it, the CRISPR companies are approaching it as such.

Ive heard many people say buckle up because there will be a trough of disillusionment that has to be traversed before it can become a clinical reality, Sontheimer says. But the potential payoff is so clear, that there will be enough staying power if and when that comes.

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CRISPR's breakthrough problem | February 13, 2017 Issue - Vol. 95 ... - The Biological SCENE

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People get test results, have more tests done at Live Healthy 2017 Health Fair – Grand Island Independent

February 12th, 2017

AURORA Live Healthy 2017 Health Fair was supposedly a one-day event that drew a number of health care providers and visitors to the Ag Building at the Hamilton County Fairgrounds.

But some people prepared for the Saturday event well in advance by attending one of the three Memorial Health Clinics in January and February to get a low-cost blood draw, according to Tina Hunt, marketing director for Aurora Community Health. Hunt said people then could go to Saturdays health fair to pick up their results, or they could go to Memorial Healths online portal.

Its a big thing for people to pick up their results, Hunt said.

She provided a folder that showed blood draw test results for white and red blood counts.

The folder also provided explanations for what each test might possibly mean. For example, a low amount of red blood cells could indicate anemia.

The blood draw also included explanations for five cholesterol and triglycerides tests, including explanations of what each means and when it might be advisable to consult a physician.

Several tests look at both kidney and liver function.

Its a complete blood panel, but also we do TSH (thyroid stimulating hormone), Hunt said. Theres some extra thyroid functioning tests with it. People may want to do the extra A1C, which is for diabetes. And then we also have the PSA (prostate-specific antigen). Its really a complete panel plus.

Both the A1C and PSA tests came with an additional cost.

People could also schedule appointments to get three types of cardiovascular tests performed by the Bryan Health mobile screening unit. That was a popular attraction.

Bryan Health was not the only health-care provider from outside Aurora to be at Saturdays health fair. CHI Health St. Francis had a visually arresting booth that showed the health pink lung of a non-smoker next to a blackened, shriveled lung for a smoker.

The Central District Health Department, based in Grand Island, but covers Hall, Hamilton and Merrick counties, also had a booth in the Ag Building. Senior living and assisted-living centers near Aurora were also at the fair. There were booths for hospice care, message therapists and an organization that sells devices for older people living at home, for use in an emergency such as a fall.

But with Memorial Community Health acting as the host for its own event, it was no surprise they had the most booths.

We have 14 booths, Hunt said, who noted that those booths showed the full range of health care provided by Memorial Community Health, including:

Memorial Community Care, which provides long-term care.

The three Memorial Health Clinics, located in Aurora, Clay Center and Harvard.

East Park Villa, which provides assisted and independent living.

Memorial Community Health, the hospital.

Hunt said the health fair also allows Memorial Community Health to show off its many departments and services such as radiology, diabetes education and pulmonary rehab. She said pulmonary rehab is relatively new, with the pulmonary and respiratory therapist busy working with people who have COPD, chronic bronchitis and other respiratory ailments. She said people may be surprised at the broad range of surgeries performed at Memorial Community Health.

The health fair had a booth for its OB/GYN department. However, Hunt noted that Memorial Community Health also has a doula who provides information, as well as emotional and physical support to women before, during and after childbirth.

The health fair also included a couple fitness demonstrations and a yoga demonstration by Mardell Jasnoski.

Jasnoski said some people say they are not limber enough to do yoga but, she added, that is the very reason they should consider it. She said people can increase their flexibility through yoga.

Jasnoski said yoga also works helps people with deep breathing, as well as maintaining balance, which becomes increasingly important as people age.

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People get test results, have more tests done at Live Healthy 2017 Health Fair - Grand Island Independent

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Women’s wellness: Understanding hypothyroidism and pregnancy – The Killeen Daily Herald

February 12th, 2017

When a woman becomes pregnant, many changes occur in her body. One of those changes is in the levels of various hormones produced by the body.

In the case of thyroid-stimulating hormone (TSH), pregnant women typically produce a lower level than normal (0.44.0 milli-international units per liter). Some international guidelines recommend levels be no higher than 2.5-3. milli-international units per liter during pregnancy. When their TSH levels rise above this, they may experience subclinical hypothyroidism, or mildly underactive thyroid, which can cause a number of health problems if left untreated.

Today, Mayo Clinic researchers report that one of those results could be pregnancy loss. Researchers further suggest a course of action that could positively impact as many as 15 of every 100 pregnancies. In a study published in The BMJ, they show that treating subclinical hypothyroidism (not quite the level that would be treated in a nonpregnant woman) can reduce pregnancy loss, especially for those with TSH levels on the upper end of normal or higher.

A recent analysis of 18 studies showed that pregnant women with untreated subclinical hypothyroidism are at higher risk for pregnancy loss, placental abruption, premature rupture of membranes, and neonatal death, said Dr. Spyridoula Maraka, an endocrinologist and lead author of the study. It seemed likely that treating subclinical hypothyroidism would reduce the chance of these deadly occurrences. But we know that treatment brings other risks, so we wanted to find the point at which benefits outweighed risks.

Using the OptumLabs Data Warehouse, Maraka and her team examined the health information of 5,405 pregnant women diagnosed with subclinical hypothyroidism. Of these, 843 women, with an average pretreatment TSH concentration of 4.8 milli-international units per liter, were treated with thyroid hormone. The remaining 4,562, with an average pretreatment TSH concentration of 3.3 milli-international units per liter, were not treated.

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5 reasons you should sign up for the bone marrow registry right now – New York Daily News

February 12th, 2017

NEW YORK DAILY NEWS

Saturday, February 11, 2017, 5:00 AM

So, bless your heart, youve already signed up for your states organ donor registry. Now its time to kick your lifesaving quest up a notch and sign up for the National Marrow Donor Program, which helps match potential donors with patients fighting leukemia, lymphoma and other deadly diseases.

Here are five reasons to throw your name in the hat, if you needed some convincing:

You can join in person by stopping by a registration drive or by spending a few minutes on BeTheMatch.org. Either way, youll get a registration kit to provide a cheek swab, which the organization uses to identify tissue type and match with a patient.

People aged 18 to 44 hit the sweet spot, as theyre called upon 90% of the time the younger the donor, the smoother the recovery for both patient and donor, said Lauren Wollny, the New Jersey/New York community engagement representative for the nonprofit Icla Da Silva Foundation. The 45- to 60-year-old crowd can still sign up, albeit for a $100 tax-deductible fee that helps the nonprofit cover costs.

Bradley Cooper urges public to join bone marrow registry

Just 1 in 430 volunteers ever even get a call to begin the donation process.

Once youre identified as a match, youll submit to a blood test, physical exam and pregnancy test, all free of charge. A doctor will then recommend one of two procedures: a nonsurgical peripheral blood stem cell (PBSC) donation (75% of the time), or bone marrow donation (25%), which involves surgery and anesthesia but isnt nearly as horrifying as youve heard.

For PBSC, the most common method, youll receive an injection of the drug filgrastim for five days leading up to the donation. On the big day, youll head to a clinic or blood center to have a needle draw blood from one arm, pass it through a machine that isolates the blood-forming cells, and return the blood to the other arm voila. Depending on the size of both patient and donor, it can take four to eight hours which you might use to reflect on what a terrific thing youre doing for a total stranger.

For bone marrow donation youll head to the OR, where a doctor will siphon liquid marrow from the back of your pelvic bone with a needle. The anesthesia will keep you numb, and though you may later feel back or hip soreness, fatigue and other side effects, you should be back to your normal routine within a week.

EXCLUSIVE: Bone marrow recipients to meet FDNY donors

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This is the one that gets a really bad rap, Wollny told the Daily News. Its not as bad as people make it out to be.

All in all, the average length of the donation process from start to finish is about 20 to 30 hours over a month or two and your own personal case manager will see you through the entire thing.

People are most likely to match with someone of the same ethnic background since the tissue types used for matching are inherited and the registry is starved for donors who are black or African American, Hispanic, Hawaiian/Pacific Islander, Asian, Alaska native, Native American and multiracial. If one of those describes your ancestry, go be a hero, please.

The Fort Lee, N.J., 12-year-old was diagnosed with acute myeloid leukemia last year, undergoing several rounds of chemo and a bone marrow transplant from her mom before eventually being pronounced cancer-free. But after a relapse in November, Lopez is fighting for her life once again and desperately in need of another bone marrow transplant.

New York tries to increase organ donations to those in need

The Long Island City-based Icla Da Silva Foundation will hold combo registry drive/fundraisers for the tween in New Jersey (Fort Lee and Union City), Georgia, Texas and Florida this Sunday. If youre free and in good health, you should go.

We hope to find Briana a match, and if we find other people a match as well, fantastic, Wollny said. Its so simple to save a life if it was you, wouldnt you want someone to do that for you?

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5 reasons you should sign up for the bone marrow registry right now - New York Daily News

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Can banking baby teeth treat diabetes? – Fox News

February 12th, 2017

When she was just 11 months old, Billie Sue Wozniaks daughter Juno was diagnosed with type 1 diabetes, an autoimmune disease that affects 1.25 million people and approximately 200,000 children under age 20 in the United States.

The disease had affected several members of Billie Sues family, including her uncle, who passed away at the age of 30.

My first thought was, Her life is going to be short, the 38-year-old from Reno, Nevada recalled. The more that I learned, the more I found that many people with type 1 live longer and the treatment advances are really exciting.

While looking for treatments, Wozniak learned about encapsulation therapy, in which an encapsulated device containing insulin-producing islet cells derived from stem cells is implanted under the skin. The encapsulation device is designed to protect the cells from an autoimmune attack and may help people produce their own insulin.

After learning of the therapy through JDRF, Wozniak saw an ad on Facebook for Store-A-Tooth, a company that offers dental stem cell banking. She decided to move forward with the stem cell banking, just in case the encapsulation device became an option for Juno.

In March 2016, a dentist extracted four of Junos teeth, and sent them to a lab so her stem cells could be cryopreserved. Wozniak plans to bank the stem cells from Junos molars as well.

Its a riskI dont know for sure if it will work out, Wozniak said.

Dental stem cells: a future of possibilities

For years, stem cells from umbilical cord blood and bone marrow have been used to treat blood and bone marrow diseases, blood cancers and metabolic and immune disorders.

Although there is the potential for dental stem cells to be used in the same way, researchers are only beginning to delve into the possibilities.

Dental stem cells are not science fiction, said Dr. Jade Miller, president of the American Academy of Pediatric Dentistry. I think at some point in time, were going to see dental stem cells used by dentistson a daily practice.

Dental stem cells have the potential to produce dental tissue, bone, cartilage and muscle. They may be used to repair cavities, fix a tooth damaged from periodontal disease or bone loss, or even grow a tooth instead of using dental implants.

In fact, stem cells can be used to repair cracks in teeth and cavities, according to a recent mouse study published in the journal Scientific Reports.

Theres also some evidence that dental stem cells can produce nerve tissue, which might eliminate the need for root canals. A recent study out of Tufts University found that a collagen-based biomaterial used to deliver stem cells to the inside of damaged teeth can regenerate dental pulp-like tissues.

Dental stem cells may even be able to treat neurological disorders, spinal cord and traumatic brain injuries.

I believe those are the kinds of applications that will be the first uses of these cells, said Dr. Peter Verlander, Chief Scientific Officer for Store-A-Tooth.

When it comes to treating diseases like type 1 diabetes, dental stem cells also show promise. In fact, a study in the Journal of Dental Research found that dental stem cells were able to form islet-like aggregates that produce insulin.

Unlike umbilical cord blood where theres one chance to collect stem cells, dental stem cells can be collected from several teeth. Also, gathering stem cells from bone marrow requires invasive surgery and risk, and it can be painful and costly.

The stem cells found in baby teeth, known as mesenchymal cells, are similar to those found in other parts of the body, but not identical.

There are differences in these cells, depending on where they come from, Verlander said.

Whats more, mesenchymal stem cells themselves differ from hematopoietic, or blood-forming stem cells. Unlike hematopoietic stem cells, mesenchymal stem cells can expand.

From one tooth, we expect to generate hundreds of billions of cells, Verlander said.

Yet the use of dental stem cells is not without risks. For example, theres evidence that tumors can develop when stem cells are transplanted. Theres also a chance of an immune rejection, but this is less likely if a person uses his own stem cells, Miller said.

The process for banking stem cells from baby teeth is relatively simple. A dentist extracts the childs teeth when one-third of the root remains and the stem cells are still viable. Once the teeth are shipped and received, the cells are extracted, grown and cryopreserved.

Store-A-Tooths fees include a one-time payment of $1,749 and $120 per year for storage, in addition to the dentists fees for extraction.

For families who are interested in banking dental stem cells, they should know that theyre not necessarily a replacement for cord blood banking or bone marrow stem cells.

Theyre not interchangeable, we think of them as complementary, Verlander said.

Although the future is unclear for Junowho was born in 2008her mom is optimistic that shell be able to use the stem cells for herself and if not, someone else.

Ultimately, however, Wozniak hopes that if dental stem cells arent the answer, there will be a biological cure for type 1 diabetes.

I hold out hope that somewhere, someone is going to crack the code, she said.

Julie Revelant is a health journalist and a consultant who provides content marketing and copywriting services for the healthcare industry. She's also a mom of two. Learn more about Julie at revelantwriting.com.

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Can banking baby teeth treat diabetes? - Fox News

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Medical schools steadily improve clinical care with research – Crain’s Detroit Business

February 12th, 2017

Crain's Detroit Business

Medical schools steadily improve clinical care with research
Crain's Detroit Business
... study on whether intravenous delivery of nutrients into the first part of the intestine or stomach will reduce eating and improve weight-related conditions to Wayne State's novel gene therapy research for blinding eye disease, which affects 100,000 ...

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Medical schools steadily improve clinical care with research - Crain's Detroit Business

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The impact of RABL2B gene (rs144944885) on human male infertility in patients with oligoasthenoteratozoospermia … – UroToday

February 11th, 2017

Male infertility is a multifactorial disorder with impressively genetic basis; besides, sperm abnormalities are the cause of numerous cases of male infertility. In this study, we evaluated the genetic variants in exons 4 and 5 and their intron-exon boundaries in RABL2B gene in infertile men with oligoasthenoteratozoospermia (OAT) and immotile short tail sperm (ISTS) defects to define if there is any association between these variants and human male infertility.

To this purpose, DNA was extracted from peripheral blood and after PCR reaction and sequencing, the results of sequenced segments were analyzed. In the present study, 30 infertile men with ISTS defect and 30 oligoasthenoteratozoospermic infertile men were recruited. All men were of Iranian origin and it took 3years to collect patient's samples with ISTS defect.

As a result, the 50776482 delC intronic variant (rs144944885) was identified in five patients with oligoasthenoteratozoospermia defect and one patient with ISTS defect in heterozygote form. This variant was not identified in controls. The allelic frequency of the 50776482 delC variant was significantly statistically higher in oligoasthenoteratozoospermic infertile men (p<0.05). Bioinformatics studies suggested that the 50776482 delC allele would modify the splicing of RABL2B pre-mRNA. In addition, we identified a new genetic variant in RABL2B gene.

According to the present study, 50776482 delC allele in the RABL2B gene could be a risk factor in Iranian infertile men with oligoasthenoteratozoospermia defect, but more genetic studies are required to understand the accurate role of this variant in pathogenesis of human male infertility.

Journal of assisted reproduction and genetics. 2017 Jan 30 [Epub ahead of print]

Seyedeh Hanieh Hosseini, Mohammad Ali Sadighi Gilani, Anahita Mohseni Meybodi, Marjan Sabbaghian

Department of Andrology, Reproductive Biomedicine Research Center, Royan Institute for Reproductive Biomedicine, ACECR, Tehran, Iran., Department of Genetics, Reproductive Biomedicine Research Center, Royan Institute for Reproductive Biomedicine, ACECR, Tehran, Iran., Department of Andrology, Reproductive Biomedicine Research Center, Royan Institute for Reproductive Biomedicine, ACECR, Tehran, Iran. .

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28138870

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The impact of RABL2B gene (rs144944885) on human male infertility in patients with oligoasthenoteratozoospermia ... - UroToday

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Male Contraceptives Have A Messy History And A Bright Future – Yahoo News

February 11th, 2017

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Why is contraception the burden of women? Male contraception would seem to be a much easier way of having sex for fun and not sticking a woman with the baby, but its rarely been on the minds of scientists in the past. That may be about to change, especially with the recent success of Vasalgel in a clinical trial. But why did it take so long, and why is it going so slowly?

Currently contraception takes three forms when it comes to men: Withdrawal, vascetomy, or condoms. Pulling out requires experience and control, making it less than ideal for an act all about losing control. Vasectomy works, but is, well, a rather permanent solution most people dont want to resort to. And condoms generally work, and have the bonus of helping prevent STIs.

That said, contraceptive options for women tend to be riskier, healthwise. Hormonal birth control may, depending on your genetics, increase your risk of stroke, and other side effects of the pill, especially the psychological ones, had been downplayed or even covered up for years or decades. Tubal ligation is more dangerous than vasectomy, albeit only by a small margin, and also a permanent solution where one may not be wanted. And IUDs have rare, but potentially serious, risks. Simply put, biology makes it much easier for men to use contraceptives, but historically, its been the womans job.

The main issue is that where women produce one cell a month, men crank out literally over a thousand sperm per second. That makes male birth control inherently more hit-or-miss since, despite making millions of them, you only need one to get pregnant. And, it has to be said, theres also the social aspect: Men dont get pregnant, and its easier to simply stick the woman with the responsibility and walk away. The history of birth control is littered with ugly incidents where sex without babies was seen as more important than womens health.

That doesnt mean, however, that men havent been trying, and even succeeding to some degree. The ancient Greeks mixed hemp seeds and rue in alcohol to lower sperm count, a method which worked in rat studies conducted thousands of years later. Gossypol, a polymer found in cottonseed oil used for cooking, turned out to be effective, but had a high risk of permanent infertility. And recently, the folklore that papaya seeds reduce fertility turned out to be accurate.

The problem is that the fields had several high profile failures. For example, a few months ago, Facebook had a good giggle at the idea of fragile men unable to handle the side effects of an experimental set of hormonal birth control shots. But that ignored that as the study has scaled up, it had gotten more and more reports of excessively increased libido from more than a third of study participants and 20% reporting mood disorders. That meant one of two things: The drug was riskier than previously thought, or something in the trial had gone wrong.

Shutterstock

There are, however, a host of other options. Calcium channel blockers, encouraging the immune system to attack sperm, and even an alpha blocker that simply prevents ejaculation are all out there and being tested. And noninvasive surgical options, like the aforementioned Vasalgel, which is already in human trials in India, and a treatment blasting the testicles with ultrasound to kill sperm, are also showing promise.

So whats the issue? Why is research so slow? In a word? Trust. Women have repeatedly expressed a discomfort in trusting men to be in charge of their reproductive destiny. In fact, it can even be a form of domestic violence: In 2010, 10% of men and 9% of women report theyve been the targets of reproductive coercion, in which someone is forced into a pregnancy by means of sabotaging their birth control, or being impregnated without their consent. And only recently have the courts viewed removing a condom during sex as a serious crime.

That, combined with the fears of some men that male birth control will make them less of a man, can be a difficult hurdle for some to jump. That said, men should be allowed to take more control of their reproductive destiny. And medical science finally seems ready to give them just that.

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Male Contraceptives Have A Messy History And A Bright Future - Yahoo News

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Women in Data Science conference highlights female participation in male-dominated field – Daily Free Press (subscription)

February 11th, 2017

Daily Free Press (subscription)

Women in Data Science conference highlights female participation in male-dominated field
Daily Free Press (subscription)
Later in the day, Caroline Uhler, a professor at MIT's Institute for Data, Systems, and Society, shared her research on weather forecasting models and her work with genetics. Audience members listened, taking notes on Uhler's data-driven research and ...

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Women in Data Science conference highlights female participation in male-dominated field - Daily Free Press (subscription)

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