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Aging and Heart Research Lead Station Science Today – Space Fellowship

Expedition 52 explored the aging process in space today and measured the lighting conditions on the International Space Station. The crew is also getting spacesuits ready for an upcoming Russian spacewalk.

Flight Engineer Peggy Whitson swapped out stem cell samples today inside the Microgravity Science Glovebox for the Cardiac Stem Cells study. The experiment is researching spaceflights effect on accelerated aging and may provide a treatment for heart disease on Earth. Scientists are observing the stem cells in space to determine their role in cardiac biology and effectiveness in tissue regeneration.

Whitson also set up light meters to measure the intensity and color of new LED (light-emitting diode) light bulbs installed in the station. The data is being collected for the Lighting Effects study to determine how the new lights affect crew sleep, circadian rhythms and cognitive performance.

NASA astronaut Jack Fischer checked out Russian Orlan spacesuits with Commander Fyodor Yurchikhin this morning. The spacesuit maintenance work is doing being done ahead of a Russian spacewalk planned for later this year.

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U team discovers ‘powerhouse’ new treatment in fight against deadly skin disease – Southernminn.com

Jonathan Pitre is a teenager who loves to write science fiction as an escape from the painful disease that causes his body to be coated with wounds.

But the breakthrough bone-marrow transplant he just received at the University of Minnesota is anything but fantasy.

A decade after performing the worlds first bone marrow transplants to treat epidermolysis bullosa a rare and potentially fatal skin disease university researchers believe they have discovered a powerhouse new formula that advances their research, helps the body grow new skin and will allow patients such as Pitre, 17, to live longer, less painful lives.

Its really not miraculous. It certainly isnt science fiction, said Dr. Jakub Tolar, director of the Us stem cell institute and the world leader in transplant therapies for EB. Its based on the hard work of our predecessors. You accomplish something and then you use that knowledge to enhance the next step and the next step.

When they conducted the first transplants using donor bone marrow and umbilical cord blood in 2007, Tolar and colleagues were trying to produce a collagen that binds skin together and is lacking in EB patients. But they had little certainty about the types of cells that would work best.

Since then, research discoveries have allowed them to home in on mesenchymal stem cells, which they believe are uniquely good at bullying their way into the body and producing the missing collagen.

This is the first time ever, that I know of, when you are infusing them with the goal that these cells will stay, Tolar said. They will graft into the skin, set up shop there. Its as if these mesenchymal stem cells are coming home.

The doctors have also focused on transplants involving bone marrow from relatives, which is more familiar to the body and less likely to be rejected by the recipients.

A transplant like Jonathans occurs in a one-two punch. After receiving radiation and chemotherapy treatments to suppress the immune system, the patient receives an infusion of hematopoietic blood stem cells from a donor. Their job in this procedure is to give the patient a new immune system that wont reject the donors mesenchymal cells when they are transplanted later.

Since the U received federal approval last fall to offer the treatment experimentally, seven patients have undergone the procedure.

Tolar said all seven are progressing though Jonathan needed a second transplant this spring because the first one failed to knock out his old immune system.

Jonathan suffered an infection after his most recent transplant, which forced him to return to the hospital this month with high fevers and blisters on his face and mouth. Even so, Jonathans mother, Tina Boileau, said she has been taking pictures since the latest transplant to document the progress for her son, whose back is covered with wounds but for a healthy spot on his right shoulder blade.

Theyre actually in scabs, a sign of healing, said Boileau, who was the bone marrow donor for her sons transplant. Which Ive never seen before.

10 patients died

EB afflicts about one in every 30,000 to 50,000 people, though some forms are more severe than others. While it is known largely for the grotesque skin wounds it causes, the disease is often fatal because it leads to severe infections or skin cancers. It can also create internal wounds to the patients digestive tract, which impairs eating.

The desperation of children with the disorder and their families compelled the first transplants at the university in 2007. Even using the old approach, about two-thirds of patients saw improvements, but 10 of the first 30 recipients died from their diseases or complications of treatment.

The Us latest success with mesenchymal stem cells might end up being an incremental step. Earlier this year, Tolar and his colleagues published research showing success in an even more advanced therapy: laboratory testing using gene editing that can reprogram the patients cells to produce healthy skin cells and tissue.

Further successes could lead to clinical trials in which a patients own dysfunctional cells would be reprogrammed, preventing the need for chemotherapy and the replacement of their immune systems.

Before they came to the U, Boileau said, her son had run out of options. Managing his pain, once possible with over-the-counter Advil, had come to require opioid painkillers such as methadone. That made him groggy and complicated his already awkward life at school back home in Ottawa. Jonathan wasnt even able to eat lunch in the school cafeteria for fear of being accidentally bumped and suffering fresh wounds.

Then the Canadian government approved funding to make him his countrys first recipient of an experimental bone marrow transplant for EB. And his home community rallied to support the family. Among other things, he has visited with pro hockey players from the Ottawa Senators, which also issued a contract adding him to their scout staff.

After seeing the pain her son has endured, Boileau said shell never complain about a blister from new shoes. She marvels at his optimism and his use of science fiction reading and writing to escape.

Inspired by the success of Christopher Paolini, who wrote the acclaimed Eragon science fiction novel as a teen, Jonathan has resolved to write his own science fiction book about a teen who develops the ability to overcome EB. The project resulted in long visits and e-mail exchanges between Tolar and his patient about medicine and physics, because Jonathan wants his story grounded in reality.

Theyre almost soul mates, Boileau said.

Tolar said he enjoys the intellectual relationship and that his patient is providing an example of hope and teaching others about the disease: He may be the only person [who] can bring this kind of view to others, Tolar said.

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Adult Stem Cells Save Woman Ravaged by Lupus, Now She Can be … – LifeNews.com

Today the Charlotte Lozier Institute announced the release of its latest testimonial video at StemCellResearchFacts.org, a project of the Washington, D.C.-based research and policy group. The video revisits Jackie Stollfus, a lupus survivor whose story was first told in a video released in 2014.

Diagnosed at the age of 21 with systemic lupus, an autoimmune disease with no known cure, Stollfus endured years of debilitating symptoms that did not respond to medication before undergoing a transplant of her own bone marrow stem cells. Seven years later, she is healthy, active, and has been able to start a family. Adult stem cells saved my life, gave me a chance to have a life, gave me that chance to be a mom, she says.

Dr. David Prentice, Vice President and Research Director of the Charlotte Lozier Institute and an international expert on stem cells, hailed the new video, saying:

Follow LifeNews.com on Instagram for pro-life pictures.

Autoimmune diseases are notoriously challenging to treat, which makes Jackie Stollfuss recovery that much more striking. As this video shows, adult stem cells are the gold standard for stem cells when it comes to patient-centered science. Jackies story is only the latest example of innovation using adult stem cells. These non-controversial cells have led to validated healing in FDA-approved studies and peer-reviewed publications for patients with various diseases and conditions. Derived from bone marrow, umbilical cord blood, and other ethical sources, they have already been used to help over one million suffering patients around the globe.

Charlotte Lozier Institute President Chuck Donovan praised Congressional efforts to prioritize NIH funding for the most promising research:

The initial successes for these innovative therapies must be followed up with expanded resources to bring more treatments to the clinic and the bedside. The bipartisan, aptly-named Patients First Act (H.R. 2918) introduced by Rep. Jim Banks and Rep. Dan Lipinski is a good example of how policymakers can advance cutting-edge medicine. It directs resources for stem cells where they will do the most good for patients.

StemCellResearchFacts.org, a project of the Charlotte Lozier Institute, was established in 2009 to facilitate and form a worldwide community dedicated to helping individuals, patients and families discover, learn and share the latest advances in adult stem cell research. To that end, the website has published 16 video testimonials backed by peer-reviewed published science. These testimonials feature patients who have undergone successful therapies for a variety of conditions including autoimmune diseases, cancer, spinal cord injury, heart disease, and more using adult stem cells. They also convey the testimony of doctors and researchers on the merits of these treatments.

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Making Babies, No Sex Necessary – The Atlantic

In the future, when a couple wants to reproduce, they will not make a baby in a bed or in the backseat or a car, or under a Keep Off the Grass sign, says Henry Greely, the director of the Center for Law and the Biosciences at Stanford Law School.

Instead, they will go to a clinic. Using stem cells from the couples skin or other non-reproductive organs, scientists will be able to make eggs and sperm, which will be combined into embryos. Each of those embryos will have its own gene sequence, Greely says. The parents will be asked: What do you want to know about these embryos? And theyll be told.

Twenty or 30 years from now, parents will be able to screen their potential kids for genetic abnormalities, pre-disposal to disease, sex, and even cosmetic features like hair, eye, and skin color, Greely claims. The new way of baby-making will save women the pain of going through fertility treatments, he says, and it will prevent disease, save health-care costs, and give non-traditional families more chances to have children. If this reproductive future comes to pass, it will also come with a tangle of moral, legal, and medical questionsones that wont be easy to resolve, despite what Greely may think.

When Greely tells people about his theorywhich is the subject of his 2016 book, The End of Sex and the Future of Human Reproductionthey tend to say, This is Gattica, or this is Brave New World, he said during an interview with the New York Times reporter Carl Zimmer on Monday at the Aspen Ideas Festival, which is co-hosted by the Aspen Institute and The Atlantic. Greely is skeptical of this argument. This is not designer babies. This is not super babies. This is selecting embryos, he said.

Greely gets some of his confidence from the limits of science. Geneticists likely wont be able to predict kids behavioral traits, he said, like their aptitude for math or agility on a sports field. But they may be able to anticipate some traits, like intelligence, in broad strokes. Being able to tell parents that this embryo has a 60 percent chance of being in the top half [of their school class], this embryo has a 13 percent chance of being in the top 10 percentI think thats really possible, he said.

Scientists have been screening embryos using a process called preimplantation genetic diagnosis, or PGD, for two and half decades, Greely said. This allows for the detection of some genetic diseases, as well as determining the sex of the embryo. Up until now, it has been expensive and arduous, but with new technologyincluding the expanded use of stem cellsit will become easy, he said. The people most likely to lead the way on easy PGD are those with fertility trouble, he argues, or those who cant have their own biological kids, including same-sex couples. For these people, the process seems to be a clear potential win: Once hopeless, they may soon be able to have biological children of their own.

But if the process does indeed advance in the way Greely predicts, it will come with big ethical challenges. Safety is a big issue, he said. Coercion is a big issue: Will you be forced to do this? No matter how easy PGD becomes, it will always be expensive, meaning that babies from rich families would gain even more advantages over other people before they leave the womb. The procedure also challenges the disability-rights movement, Greely pointed out: It implicitly suggests that some traits, and thus some people, are preferable to others.

Theres very little about our modern lives that a God from 3000 years ago would have expected.

Some critics may also claim this process is against Gods will, Greely added. I dont have a lot of confidence in the intellectual strength of that argument, but I think it has a lot of visceral support.

Despite Greelys skepticism, this seems to be the greatest potential objection to a world of skin-cell babies and intensive genetic screening: It assumes that the creation of life is a matter of pipettes and petri dishes, not something greater. While the widespread use of contraceptives has largely divorced sex from procreation, this process would represent the final severing. As Greely pointed out, the very meaning of sex would change. Most people have sex and it doesnt result in a baby, he said. They do it because they like it. They do it as a token of love. They do it because theyre forced to. They do it to make money. Pleasure, ultimately, will be a main driver of sex, he added.

For the many peoplereligious or notwho believe that life is not ultimately a matter of science, the world of easy PGD may seem disorienting, even morally disturbing. But Greely didnt think religious or moral arguments could persuade someone like him, or society more broadly, that easy PGD isnt a good idea.

If you, coming from a Catholic background, try to convince me, coming from a non-Catholic background … that wouldnt work for me, he said. I need a more intellectual argument than one based on my faith or the tablets brought down from the mountain for me say this. Theres very little about our modern lives thats natural or what a God from 3000 years ago would have expected or wanted, including all of modern medicine.

As head-spinning as these theoretical ethical challenges are, perhaps easy PGD wont be as common as Greely thinks. After all, he joked, were never going to get rid of teenagers in the back seat of a car.

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Making Babies, No Sex Necessary – The Atlantic

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What makes stem cells into perfect allrounders – Phys.org – Phys.Org

June 27, 2017 Just a few days old embryonic cell clusters: with functional Pramel7 (left), without the protein (right) the development of the stem cells remains stuck and the embyos die. Credit: Paolo Cinelli, USZ

Researchers from the University of Zurich and the University Hospital Zurich have discovered the protein that enables natural embryonic stem cells to form all body cells. In the case of embryonic stem cells maintained in cell cultures, this allrounder potential is limited. Scientists want to use this knowledge to treat large bone fractures with stem cells.

Stem cells are considered biological allrounders because they have the potential to develop into the various body cell types. For the majority of stem cells, however, this designation is too far-reaching. Adult stem cells, for example, can replace cells in their own tissue in case of injury, but a fat stem cell will never generate a nerve or liver cell. Scientists therefore distinguish between multipotent adult stem cells and the actual allrounders – the pluripotent embryonic stem cells.

Epigenetic marks determine potential for development

Differences exist even among the true allrounders, however. Embryonic stem cells that grow in laboratory cell cultures are in a different state than the pluripotent cells found inside the embryos in the first days of development. In a study in the journal Nature Cell Biology, researchers led by Paolo Cinelli of the University Hospital Zurich and Raffaella Santoro of the University of Zurich have now demonstrated the mechanism by which natural allrounders differ from embryonic stem cells in cultures.

At the center of their discovery is a protein called Pramel7 (for “preferentially expressed antigen in melanoma”-like 7) found in the cells of embryonic cell clusters that are just a few days old. This protein guarantees that the genetic material is freed from epigenetic marks consisting of chemical DNA tags in the form of methyl groups. “The more methyl groups are removed, the more open the Book of Life becomes,” Cinelli says. Since any cell of the human body can develop from an embryonic stem cell, all genes have to be freely accessible at the beginning. The more a cell develops or differentiates, the stronger its genetic material is methylated and “sealed closed” again. In a bone cell, for example, only those genes are active that the cell requires for its function, the biochemist explains.

Protein is responsible for perfect pluripotency

Despite its short action period of just a few days, Pramel7 seems to play a vital role: When the researchers headed up by Cinelli and Santoro switched off the gene for this protein using genetic tricks, development remained stuck in the embryonic cell cluster stage. In the cultivated stem cells, on the other hand, Pramel7 is rarely found. This circumstance could also explain why the genetic material of these cells contains more methyl groups than that of natural embryonic cells – the perfect allrounders, as Cinelli calls them.

Using the stem cell function to regenerate bone tissue

His interest in stem cells lies in the hope of one day being able to help people with complex bone fractures. “Bones are great at regenerating and they are the only tissue that does not build scars,” Paolo Cinelli says. The bone stumps must be touching, however, in order to grow together. When a bone breaks in multiple places and even through the skin, for example, in a motorcycle accident, the sections of bone in between are often no longer usable. For such cases, a bone replacement is required. His team is studying carrier materials that they want to populate with the body’s own stem cells in the future. “For this reason, we have to know how stem cells work,” Cinelli adds.

Explore further: New tools to study the origin of embryonic stem cells

More information: Urs Graf et al, Pramel7 mediates ground-state pluripotency through proteasomalepigenetic combined pathways, Nature Cell Biology (2017). DOI: 10.1038/ncb3554

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Large-scale Production of Living Brain Cells Enables Entirely New Research – Laboratory Equipment

Important pieces of the puzzle to understand what drives diseases such as Alzheimer’s and Parkinson’s are still missing today. One crucial obstacle for researchers is that it is impossible to examine a living brain cell in someone who is affected by the disease. With the help of a new method for cell conversion, researchers at Lund University in Sweden have found a way to produce diseased, aging brain cells on a large scale in a cell culture dish.

After performing a biopsy on the patient, the skin cells are transformed into brain cells that effectively imitate the disease and the age of the patient. The fact that the cells can now be produced in large quantities enables researchers to carry out a series of experiments that were previously not possible.

A few years ago, Malin Parmar’s research team was one of the first in the world to convert human skin cells directly into brain cells without passing the stem cell state. The discovery shocked the researchers and was perceived as almost impossible. The team is now approaching a point where the discovery is about to bear fruit on a wide scale. By following a new method that involves slightly changing the genetic code that triggers cell conversion, the researchers were able to multiply the production of disease-specific brain cells.

“Primarily, we inhibited a protein, REST, involved in establishing identity in cells that are not nerve cells. After limiting this protein’s impact in the cells during the conversion process, we’ve seen completely different results. Since then, we’ve been playing around with changing the dosage of the other components in the previous method, which also proved effective. Overall, the efficiency is remarkable. We can now generate almost unlimited amounts of neurons from one skin biopsy”, says Malin Parmar, professor of developmental and regenerative neurobiology at Lund University.

The increase in production will have far-reaching effects. The new volumes enable research projects that were simply not viable before. Among other things, it opens up research areas linked to new drug testing, the establishment of more accurate disease models and the development of diagnostics to detect the diseases at an earlier stage.

The new cells are not only able to imitate the disease but also the patient’s age. By studying the cell in the culture dish, the researchers can now monitor the mechanisms of the disease in an “old” brain cell over time. Neurodegenerative diseases are commonly referred to as “aging brain diseases” and in order to understand them, we must better appreciate how the age specifically affects the course of the disease. The Lund researchers’ discovery can hopefully contribute a crucial piece to the puzzle with regard to the connection between the onset of disease and cell aging, something which previous research based on animal experiments and stem cells has failed to provide.

“This takes us one step closer to reality, as we can now look inside the human neurons and see what goes on inside the cell in these diseases. If all goes well, this could fundamentally change the field of research, as it helps us better understand the real mechanisms of the disease. We believe that many laboratories around the world would like to start testing on these cells to get closer to the diseases”, says Johan Jakobsson, leader of the molecular neurogenetics research group at Lund University.

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Is doubling our life span desirable? – Price Sun Advocate

The times, they are a-changing.

Since Gregor Mendel unwittingly became the father of genetics by writing down his botanical observations, we have been progressing along swimmingly in our understanding and application of biology.

In the past few years, we ourselves have made some measured leaps forward in the field of biotechnology, some small someless so. Yet with the monumental achievements we have made thus-far from the advent of vaccines to our understanding of how our bodies age and degenerate, we have yet to make that quantum leap forward. That quantum leap may itself not be that far off and if anything is a good indicator of that its observable in the nature of the biotech we are currently developing.

With any huge leap forward, however, come new challenges and a slew of new questions that desperately need to be answered.

This next step in our journey isnt quite like when we eradicated major diseases or began transplanting organs because it isnt about extending human life a mere few additional years. We are taking about a doubling in the years a human may live. Thats right, double.

Now, before you write this off as sci-fi or wishful thinking, let me walk you through exactly what breakthroughs are currently occurring. It all has to do with CRISPR gene-line editing and 3-dimensional printing.

We are at the point where we can take normal somatic cells like the ones from your skin, coax them back into stem cells then re-engineer them into just about any type of cells we want. This means shortly we will be able to take skin cells and make them into heart tissue, or liver, or pancreatic or any number of different ones.

Next, the advances in 3-dimensional printing may shortly be able to take your newly minted cells and print them onto a blank scaffolding in the shape of just about any organ you may need.

Think of that: if you need a new heart it could be as simple as scratching some skin from your arm, reprogramming the cells and then printing you a whole new organ. Not a transplant from a donor, your own cells. This means no rejection and no waitlists. When an organ fails we replace it, again and again and again.

What is to become of a human race that is capable of living seemingly without end? This brings up some serious questions that would have to be answered quickly.

For starters, we see that the current population growth of our species is unsupportable as we resist green energies and advanced farming methods. If humans were to begin to live twice as long or longer we must figure out what we are going to do.

Now the radicals would suggest we simply control the populations but I dont believe that is necessary or even morally right. All we must do is increase our carrying capacity. I must admit that was not my own musing but one my father suggested to me.

If we are able to increase how much food and energy we produce without damaging the planet there is virtually no limit to how many humans can live at once. But the question is, will we resist it as we are now? Will the prospect of living healthily well over a century spur us to begin to accept scientific consensus? Or will we continue down our current path of selfishness and greed? Only time will tell.

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CRISPR gene-editing reverses Huntington’s Disease in mice – CGTN America (blog)

There may be new hope for those suffering from a fatal brain disorder called Huntingtons Disease. Recent research at Emory University, using a groundbreaking gene editing tool called CRISPR-Cas9, has provided new insight into how the disease works, and possible ways to reverse its cruelest effects.

Huntingtons Disease is a genetically inherited condition that leads to nerve cell destruction in the brain. Symptoms, which usually appear in mid-life, include uncontrolled muscle movement, balance issues, mood swings and cognitive decline. Though rates and recording vary from country to country, approximately 30,000 in the U.S. currently suffer from the disease.

While there is no known cure for Huntingtons, a recent study by Chinese scientists at Emory University in Atlanta, Georgia is showing promise. Early results suggest possible treatments for the disease and a path to preventing its occurrence in the first place.

The research is part of an ongoing medical collaboration between the U.S. National Institute of Health (NIH) and the National Natural Science Foundation of China. Under this program, both the U.S. and China contribute funds and scientists for research in both countries.

Emory School of Medicine

Using the revolutionary gene editing technique known as CRISPR-Cas9, researchers at Emory were able to reverse the effects of Huntingtons in test mice.The mice had been genetically modified to carry a human version of the hungtingtin gene that causes the disease. While considered essential for nervous system development in early life, a mutated huntingtin gene can also produce toxic proteins that cause neural generation.

After nine months, when the mice developed the animal version of Huntingtons Disease, researchers used CRISPR-Cas9 to replace the mutant gene with a normal one and then reintroduce the repaired DNA into mice.

Weeks after treatment, the brain-damaging proteins had almost disappeared and motor functions of the mice dramatically improvedthough not to the same level in healthy control mice in which Huntingtons hadnt been induced.

While the results show promise for future human trials involving humans, clinical trials remain a long way off. The long term effectiveness and safety of CRISPR-Cas9 is still under review.

The studys senior author Dr. Xiao-Jiang Li, PhD is optimistic. The findings open up an avenue for treating Huntingtons as well as other inherited neurodegenerative diseases, although more testing of safety and long-term effects is needed, said Xiao-Jiang.

In addition to developing a treatment for victims of Huntingtons, the Sino-U.S. research group hopes to develop ways to reduce the risk for people who are genetically predisposed to developing Huntingtons.

Last year, the same group of Emory researchers had shown they could delete the huntingtin gene in mice older than four months without any known adverse effects. Younger mice without this gene developed fatal pancreatitis. The findings suggest it may someday be possible to safely shut off the gene in adult humans, as well.

Full results of the groups research was published June 19, 2017 in the Journal of Clinical Investigation.

MORE ON CRISPR:

For the second time in a year, doctors in China have used the CRISPR-Cas9 gene editing technique for the treatment of cancer. It is also only the second time CRISPR-Cas9 has been used in human tria

CRISPR-Cas9 is a recently developed gene editing technique that has received worldwide attention because of its relative technical simplicity and wide applications. It is being used in research throughout the world in areas including agriculture, creating new and effective drugs, as well as treating a wide array of genetic disorders.

Defective genes can cause disease. Researchers can use CRISPR Cas-9 like a surgeon uses a scalpelslicing out bad DNA from a damaged genome. Molecular biologists can also transplant normal genes into cellsreplacing damaged or mutated DNA with a new sequence assembled in a lab.

A visualization from the McGovern Institute at MIT explains the science and approach of CRISPR-Cas9.

As the technology advances, scientists hope to someday use CRISPR-Cas9 to create gene therapies that can prevent other inherited diseases, including sickle-cell anemia, Parkinsons disease and cancers that appear to have a genetic component like colon cancer.

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CRISPR gene-editing reverses Huntington’s Disease in mice – CGTN America (blog)

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Spark Therapeutics CEO talks drug pricing, gene therapy & his Philadelphia roots – Philadelphia Business Journal


Philadelphia Business Journal
Spark Therapeutics CEO talks drug pricing, gene therapy & his Philadelphia roots
Philadelphia Business Journal
Spark Therapeutics CEO talks drug pricing, gene therapy & his Philadelphia roots. Jun 27, 2017, 2:23pm EDT. Industries & Tags: Health Care … Exclusive Online Tools. Research the 3+ year digital archive, and People on the Move leads database download.
Cell, gene therapies are hot. But can this startup make them safer?San Francisco Business Times

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Spark Therapeutics CEO talks drug pricing, gene therapy & his Philadelphia roots – Philadelphia Business Journal

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2017 Firestone and Golden medal, Kennedy Thesis Prize recipients honored – Stanford University News

by Arik Lifschitz on June 28, 2017 12:10 am

Firestone Medal

A recent ceremony honored 33 prize winners representing 26 departments from the three Stanford schools with undergraduate programs Humanities and Sciences; Engineering; and Earth, Energy & Environmental Sciences.

Harry Elam, senior vice provost for education, hosted the June 17 event for the class of 2017 recipients of the Firestone and Robert M. Golden medals and the David M. Kennedy Honors Thesis prizes.

The Firestone and Golden medals are awarded to the top 10 percent of theses completed in a given year. The Firestone Medal for Excellence in Undergraduate Research recognizes theses written in the social sciences, natural sciences, and engineering and applied sciences. The Golden Medal for Excellence in Humanities and Creative Arts similarly distinguishes theses in the humanities or creative projects in the fine arts. The medalists each received an engraved bronze medal, a citation and a monetary award.

The Kennedy Prize is awarded annually to the single best thesis in each of the four areas of humanities, social sciences, natural sciences, and engineering. Recipients of this award have accomplished significantly advanced research in the field and have shown strong potential for publication in peer-reviewed scholarly works. The prize was established in 2008 in recognition of history Professor David M. Kennedys long-standing mentoring of undergraduate writers and his retirement from active teaching. Winners each received an engraved plaque and a monetary award.

The 2017 Kennedy Prizes were presented by Sarah Church, senior associate vice provost for undergraduate education.

The projects conducted by the winners capture the breadth of the undergraduate experience at Stanford. They included research on such topics as gene therapy and protein engineering, computer science education, efficient matching algorithms, the history of national security and the authors of American romanticism. Other winners wrote and directed plays, crafted exceptional poems and excelled as flutists.

The awardees, their thesis titles, honors program or department, and advisers are as follows:

The David M. Kennedy Honors Thesis Prizes

Natural Sciences: Ryan Badiee, Engineering Bidirectional Regulation of Endogenous Genes, Biology, advised by Michael Lin (Neurobiology).

Engineering & Applied Sciences: Griffin Dietz, Childrens Use of Decomposition in Problem Solving as an Early Introduction to Computer Science, Computer Science, advised by James Landay (Computer Science) and Hyowon Gweon (Psychology).

Social Sciences: Whitney McIntosh, France and the Internationalization of Security: A Conceptual History of Security during the Interwar Years (1919-1933), Center on Democracy, Development, and the Rule of Law (CDDRL), advised by Stephen Stedman (CDDRL).

Humanities: Alex Torres, The Blakean Imagination in Nineteenth-Century America: Emerson, Whitman, Dickinson, English, advised by Denise Gigante (English) and Ramn Saldvar (English).

The Firestone Medal for Excellence in Undergraduate Research

Iliana Erteza Bray, Frequency Shifts and Depth Dependence of Beta Band Activity in Rhesus Premotor Cortex Perceptual Decision-Making, Electrical Engineering, advised by Krishna Shenoy (Electrical Engineering).

Marly Carlisle, We Do The Best We Can: Implementation of the McKinney-Vento Act by San Francisco Unified Social Workers, Education, advised by Jelena Obradovic (Graduate School of Education).

Maria Castro, Quantitative Analysis of White Matter Differences in Children Born Preterm and Full Term, Human Biology, advised by Heidi Feldman (Pediatrics Neonatology) and Jeffrey Wine (Psychology).

Melissa Eidman, Still Reservations: Identifying Contributors to Health on the Yurok Reservation, Human Biology, advised by Donald Barr (General Pediatrics), Gabriel Garcia (Gastroenterology and Hepatology), and Sawar Young-Tripp (California Rural Indian Health Board).

Bora Erden, Choice-Predictive Activity in the Macaque Premotor and Motor Cortex, Symbolic Systems, advised by William Newsome (Neurobiology) and James McClelland (Psychology).

Kathryn Evans, Using Viral Tools to Dissect Neural Circuits: Exploring the Nigrostriatal and Claustrocingulate Pathways in Mice, Biology, advised by Karl Deisseroth (Bioengineering).

Andrea Fisher, Genomic Analysis of Southeast Asian and Sahul Ancestry, Biology, advised by Marcus Feldman (Biology) and Richard Klein (Biology).

Zi Yang Kang, Strategy-Proof Bilateral Trade, Mathematics, advised by Jan Vondrak (Mathematics).

Sophia Laurenzi, The Gray Matter of Young Adulthood: Neuroscience, Social Trends, and Justice Reform, Science, Technology, and Society (STS), advised by Angela Garcia (Anthropology).

Lauren Newby, From Zero to Sixty: Explaining the Proliferation of Shia Militias in Iraq after 2003, Center for International Security & Cooperation (CISAC), advised by Martha Crenshaw (CISAC).

Nghia Nguyen, A Behavioral System for Imaging Prefrontal Cortex Activity in Freely Behaving Mice, Biomechanical Engineering, advised by Liqun Luo (Biology) and Ovijit Chaudhuri (Mechanical Engineering).

Brett Parker, Election or Appointment? A Quantitative Study of the Effects of Judicial Selection Method on Judicial Voting in Criminal Procedure Cases, Political Science, advised by David Brady (Political Science).

Indira Puri, On the Probability of Receiving a Top Choice Match, Economics, advised by Jonathan Levin (Economics and Graduate School of Business).

Daniel Sanchez-Ordonez, International Monetary Policy Spillover in Colombia: An SVAR Analysis, Economics, advised by John Taylor (Economics).

Lydia Tam, Identifying the Cellular Origin and Enzymatic Mechanism of Activity-regulated Neuroligin-3 Secretion, Biology, advised by Michelle Monje-Deisseroth (Neurology).

Eileen Williams, Interhemispheric Amygdala Connectivity Across Puberty and its Relation to Depressive Symptomatology, Psychology, advised by Ian Gotlib (Psychology).

Ethan Williams, Sycamore Knoll: A Wave-Planed Pop-up Structure in a Sinistral-oblique Thrust System, Southern California Continental Borderland, Geophysics, advised by Simon Klemperer (Geophysics).

Cristian Zanoci, Entanglement and Transport Properties of Non-Equilibrium Steady States of 1-D Quantum Systems, Physics, advised by Patrick Hayden (Physics) and Brian Swingle (UMD Physics).

Yuan Zhang, Bioactive Lipids Enhance Cardiomyocyte Differentiation from Human Induced Pluripotent Stem Cells, Biology, advised by Sean Wu (Medicine).

The Robert M. Golden Medal for Excellence in the Humanities and Creative Arts

Surabhi Balachander, Our Color in the Fields: Exploring the Intersections of Agriculture and Race in American Literature, Comparative Studies in Race and Ethnicity (CSRE), advised by Michele Elam (English) and Solmaz Sharif (English).

Madelaine Bixler, Writer/Director: In Volution, Theater and Performance Studies (TAPS), advised by Leslie Hill (TAPS) and Cherrie Moraga (TAPS).

Talia Charme-Zane, Freaky Forests, Gay Princes, and Guilty Children: A Queer Reading of Sondheims Into The Woods, Feminist, Gender, and Sexuality Studies, advised by Sianne Ngai (English).

Joshua De Leon, This Brown Body is a Vessel, Arts Institute, advised by Michele Elam (English), Solmaz Sharif (English), and Whitney Lynn (HIA).

Michael Gioia, The Revolutionary Priest: An Intellectual Biography of Claude Fauchet, History, advised by Keith Baker (History) and Dan Edelstein (French and Italian).

Gabriella Johnson, Re-Reading the Mulatta: A Black Feminist Approach to Understanding the Mulatta in 21st Century Literary Production, African and African American Studies, advised by Michele Elam (English) and Alvan Ikoku (Comparative Literature).

Jacqueline Langelier, Photography, Art and Art History, advised by Gail Wight (Art & Art History).

Quyen Nguyen, Danh V: Ghost Work, Art and Art History, advised by Alexander Nemerov (Art & Art History).

May Peterson, Venantius Fortunatus as Auctor of the Sacred: From Material to Ethereal in Sixth-Century Gaul, Classics, advised by Grant Parker (Classics) and Bissera Pentcheva (Art & Art History).

Al Yuen, Flute Performance, Music, advised by Alexandra Hawley (Music).

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2017 Firestone and Golden medal, Kennedy Thesis Prize recipients honored – Stanford University News

Recommendation and review posted by simmons

Gene Mutation Linked to Retinitis Pigmentosa in Southwestern US Hispanic Families – Texas Medical Center (press release)

Thirty-six percent of Hispanic families in the U.S. with a common form of retinitis pigmentosa got the disease because they carry a mutation of the arrestin-1 gene, according to a new study from researchers at The University of Texas Health Science Center at Houston (UTHealth) School of Public Health.

Retinitis pigmentosa is a group of rare, genetic eye disorders in which the retina of the eye slowly degenerates. The disease causes night blindness and progressive loss of peripheral vision, sometimes leading to complete blindness. According to Stephen P. Daiger, Ph.D., senior author of the study, an estimated 300,000 people in the U.S. suffer from the disease, which gets passed down through families.

In the study published recently in Investigative Ophthalmology & Visual Science, UTHealth researchers found that in a U.S. cohort of 300 families with retinitis pigmentosa, 3 percent exhibited a mutation of the arrestin-1 gene. However, more than 36 percent of Hispanic families from the cohort exhibited the arestin-1 mutation and they all came from areas in the Southwestern U.S., such as Texas, Arizona and Southern California.

When I started studying retinitis pigmentosa in 1985, we set out to find the one gene that causes the disease. Thirty-three years later, weve found that more than 70 genes are linked to retinitis pigmentosa, said Daiger, a professor in the Human Genetics Center and holder of the Thomas Stull Matney, Ph.D. Professorship in Environmental and Genetic Sciences at UTHealth School of Public Health.

Some of the genes that cause retinitis pigmentosa are recessive, which means two mutations are required, and some are dominant, which means you only need one mutation. Arrestin-1 piqued Daigers interest because that particular mutation is dominant while all previously found mutations in the gene are recessive. This unexpected finding shows that even a single mutation in the gene is sufficient to cause the disease.

Daiger and his team have identified the genetic cause of retinitis pigmentosa for 75 percent of families in their cohort. Possible treatments for some forms of retinitis pigmentosa are being tested but are still limited. However, the speed at which companies are developing gene therapies and small molecule therapies gives reason to hope, he said. Daiger and his collaborators have begun to connect some of the patients in the retinitis pigmentosa cohort to clinical trials that treat specific genes.

I want our cohort families to know that even if there is not an immediate cure for their specific gene mutation, at this rate it wont be long until a therapy becomes available, said Daiger, who also holds the Mary Farish Johnston Distinguished Chair in Ophthalmology at McGovern Medical School at UTHealth.

UTHealth coauthors include Lori S. Sullivan, Ph.D.; Sara J. Browne, Ph.D.; Elizabeth L. Cadena; Richard S. Ruiz, M.D., and Hope Northrup, M.D. Additional co-authors are from Nationwide Childrens Hospital; Kellogg Eye Center at the University of Michigan; Retina Foundation of the Southwest; Casey Eye Institute at Oregon Health and Science University; Vanderbilt University and the Department of Molecular and Human Genetics at Baylor College of Medicine.

Support for the study, titled A novel dominant mutation in SAG, the arrestin-1 gene, is a common cause of retinitis pigmentosa in Hispanic families in the Southwestern United States, was provided by the William Stamps Farish Fund and the Hermann Eye Fund.

Additional support was provided by the National Institutes of Health (EY007142, EY009076, EY011500, EY010572 and K08-EY026650), a Wynn-Gund TRAP Award, the Foundation Fighting Blindness, the Max and Minnie Voelker Foundation and a grant to the Casey Eye Institute from Research to Prevent Blindness.

Read more:
Gene Mutation Linked to Retinitis Pigmentosa in Southwestern US Hispanic Families – Texas Medical Center (press release)

Recommendation and review posted by simmons

It’s time for baseball to allow the use of PEDs – Fort Worth Star Telegram (blog)


Fort Worth Star Telegram (blog)
It's time for baseball to allow the use of PEDs
Fort Worth Star Telegram (blog)
A former Major League Baseball trainer is fairly certain that ballplayers are using steroids again. That's if they ever really stopped, the trainer, who worked for more than 10 seasons with a big league ballclub, recently told me. It wouldn't

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It’s time for baseball to allow the use of PEDs – Fort Worth Star Telegram (blog)

Recommendation and review posted by Bethany Smith

Advanced Cancer Drug Study Highlights Need for Novel Approaches – Technology Networks

Mohammad Alyamani, Ph.D., with lab technician Yoon-Mi Chung. Credit: Cleveland Clinic

Cleveland Clinic researchers have shown for the first time how a class of advanced prostate cancer drugs are processed in the body and how their anti-tumor activity might change depending on how they are metabolized. Their pre-clinical findings, just published in Cell Chemical Biology, may lay the foundation for improving therapies for treatment-resistant, aggressive prostate cancer.

Next-generation anti-androgens are potent drugs that work by cutting off the prostate tumors supply of androgens (male hormones), which fuel prostate cancer. The drugs, used in patients whose cancer has become resistant to hormone deprivation therapy, have been shown to improve survival in men with metastatic disease. Unfortunately, prostate tumors eventually become resistant to these drugs, highlighting the need for new therapies.

Despite an array of improved treatment options that have become available over the past decade, prostate cancer remains the second leading cause of cancer mortality in men in the United States. There are few therapeutic options for men whose cancer has become resistant to all therapies, said Nima Sharifi, M.D., lead author on the study. Our goal is to improve the use and role of these existing drugs and hopefully design new therapies that work better and longer.

Galeterone is a steroidal anti-androgen that was recently studied in a clinical trial. Dr. Sharifis team in the Cleveland Clinic Lerner Research Institutes Department of Cancer Biology has shown that when galeterone is metabolized, it is converted to the intermediate molecule D4G, which blocks androgen synthesis and reduces the amount of androgens available to cancer cells. A pitfall is that galeterone is also converted to another molecule that may stimulate the tumor.

Dr. Sharifi previously found that another steroidal anti-androgen drug, abiraterone, is metabolized in a similar manner. He went on to show in landmark studies that abiraterones metabolite D4A has greater anti-tumor activity than abiraterone alone and that other molecules stimulate tumor growth, suggesting that the drug should be fine-tuned to improve efficacy.

Dr. Sharifis new findings suggest that effective steroidal anti-androgens share common metabolic activities and that their metabolites should be closely examined for their effects on tumor survival. The findings may also guide medical decision making in the use of steroidal vs. nonsteroidal drugs for advanced prostate cancer.

New agents and a clearer understanding of drug mechanisms are both urgently required to improve outcomes for treatment-resistant advanced prostate cancer, said Dr. Sharifi. This work provides an important foundation that hopefully will lead to better treatment strategies for this disease.

This article has been republished frommaterialsprovided by Cleveland Clinic. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference

Alyamani, M., Li, Z., Berk, M., Li, J., Tang, J., Upadhyay, S., . . . Sharifi, N. (2017). Steroidogenic Metabolism of Galeterone Reveals a Diversity of Biochemical Activities. Cell Chemical Biology. doi:10.1016/j.chembiol.2017.05.020

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Advanced Cancer Drug Study Highlights Need for Novel Approaches – Technology Networks

Recommendation and review posted by simmons

Docs Call Hormone Therapy for Kids Risky – Newsmax.com – Newsmax

Using hormones to treat transgender children who feel that they are members of the opposite sex, which is becoming commonplace in medicine, could have serious long-term effects on children, doctors are warning in a new report.

In a paper published in the journal The New Atlantis this week, Paul Hruz of the Washington University Medical School and Lawrence Mayer and Paul McHugh of Johns Hopkins Medical School say such treatments could have serious health implications, the New York Post reports.

The paper, called Growing Pains: Problems with Puberty Suppression in Treating Gender Dysphoria, notes a recent analysis by UCLA found that about 1.4 million people in the United States identify as transgender, a growing number of whom are children.

The number of children diagnosed with gender dysphoria described by clinicians as incongruence between ones experienced/expressed gender and assigned gender has been on the rise.

A gender identity clinic for children in the United Kingdom, for instance, reported a 2,000 percent increase in referrals since 2009, with those for children under the age of 6 going from six to 32 in the same time period.

Well-meaning parents have been increasingly trying hormone suppression, which prevents sex organs in boys and girls from developing in the usual way, Hruz, Mayer, and McHugh note.

While that may allow children to postpone decisions about actual sex-reassignment surgery, the authors argue that this therapy may have real and long-term effects on childrens physical and psychological development.

Whether blocking puberty is the best way to treat gender dysphoria remains far from settled, they write, and it should be considered … a drastic and experimental measure.

2017 NewsmaxHealth. All rights reserved.

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Docs Call Hormone Therapy for Kids Risky – Newsmax.com – Newsmax

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The importance of regular blood tests – Catskill Daily Mail

Dear warriors,

Once we get treated for cancer and are in remission, we sometimes let our maintenance, such as physician visits and blood tests, lapse.

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The importance of regular blood tests – Catskill Daily Mail

Recommendation and review posted by simmons

Bone Therapeutics receives Intent to Grant Notice from European Patent Office for allogeneic bone cell therapy platform – OrthoSpineNews

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Gosselies, Belgium,26 June 2017; 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the bone cell therapy company addressing high unmet medical needs in orthopaedics and bone diseases, today announces that the European Patent Office (EPO) has notified the Company of its intention to grant a key patent covering its first-in-class allogeneic cell therapy technology.

Once granted, the patent titled, Osteogenic differentiation of bone marrow stem cells and mesenchymal stem cells using a combination of growth factors, will provide legal protection to Bone Therapeutics both for the manufacturing methods and for the distinct cell type used in its allogeneic cell therapy technology. Specifically, the patent covers methods to manufacture differentiated and biologically active osteoblastic (bone-forming) cells from bone marrow stem cells, using a specific combination of growth factors, and also covers a new class of osteoblastic cells suitable for allogeneic administration to the patient.

Bone Therapeutics will now validate the patent in several countries in the European Union, potentially allowing IP protection for its allogeneic bone cell therapy platform until 2029. Patents from the same patent family have already been granted in Japan, Australia and Singapore and applications are pending in the USA, Canada, India and South Korea. ALLOB, Bone Therapeutics most advanced allogeneic bone cell therapy product, is currently being evaluated in Phase I/IIA clinical trials for delayed-union fractures and spinal fusion, for which interim results are expected in the third quarter this year.

Thomas Lienard, Chief Executive Officer of Bone Therapeutics, commented: This notice from the European Patent Office confirms our allogeneic bone cell therapy technology is both innovative and distinctive. When granted, this European patent will significantly strengthen our IP position in the field of bone cell therapy, giving us further validation for the scientific and commercial development of our cell therapy products whilst also enhancing our position with respect to new partnerships.

Dr. Miguel Forte, Chief Medical Officer of Bone Therapeutics, further noted: Obtaining this patent is an important step in the development of our allogeneic bone cell therapy technology. It will provide a solid IP protection for our current work and for future technological advances, allowing us to continue our efforts to create patient-centric and commercially interesting bone cell therapy solutions.

About Bone Therapeutics

Bone Therapeutics is a leading cell therapy company addressing high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy products in clinical development across a number of disease areas targeting markets with large unmet medical needs and limited innovation. Our technology is based on a unique, proprietary approach to bone regeneration which turns undifferentiated stem cells into osteoblastic, or bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery. Our primary clinical focus is ALLOB, an allogeneic off-the-shelf cell therapy product derived from stem cells of healthy donors, which is in Phase II studies for the treatment of delayed-union fractures and spinal fusion. The Company also has an autologous bone cell therapy product, PREOB, obtained from patients own bone marrow and currently in Phase III development for osteonecrosis and non-union fractures.

Bone Therapeutics cell therapy products are manufactured to the highest GMP standards and are protected by a rich IP estate coveringnine patent families. Further information is available at: http://www.bonetherapeutics.com.

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in thispress release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

Josh Sandberg has been an executive search consultant focused exclusively on orthopedic and spine start-ups since 2004. He has had a tremendous impact in helping his clients avoid costly hiring mistakes by his deep industry knowledge and network. In 2010, Josh co-founded Ortho Spine Companies, which is the parent company of Ortho Spine Distributors (OSD), Surg.io and Ortho Sales Partners (OSP). OSD a searchable database that helps ease the frustration of finding orthopedic distributors throughout the country. Surg.io is the ultimate distributor toolkit that offers distributors the tools necessary to build the foundation of a scalable and highly functioning sales organization. OSP is an end-to-end solution that helps companies approach the Global Market in a cost efficient way. Our team has hundreds of years of experience and can help you navigate the many challenges present in bringing new technologies to the market.

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Bone Therapeutics receives Intent to Grant Notice from European Patent Office for allogeneic bone cell therapy platform – OrthoSpineNews

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‘He was a fighter’ says father after toddler dies of leukemia – CBC.ca

A Winnipeg toddlerwith acute myeloid leukemia has passed away after hundreds came forward to register as donors in an effort to help him.

After being diagnosed with the disease on Oct. 25, 2016, 20-month-old Tegveer Minhaswasforced to go through two rounds of chemotherapy, losing his hair and a lot of weight.

During that time, his family put out calls to the public to come forward and register bone marrow and stem cell information, in hopes that someone would be a match.

Hundreds of people in Manitoba, Ontario and Alberta were swabbed, and Tegveer was able to receive a stem cell donation, but Minhas said it didn’t work.

“After 8 months of struggle, he passed away on June 18th, early in the morning at 6 a.m.,” said his dad,Sukhbir Minhas.

Minhas saidhis family is trying to stay strong, but he admits they are having a hard time.

“He was a happy soul, he loved to go out, we took him to Clear Lake on June 4th, and I wish I knew that he would love it so much, we were planning to go back again,” Minhas said.

The hundreds of strangers who registered as donors, prayed for and even phoned the family to offer supportmeant the world to Minhas and his wife, he said.

“There was a time in the hospital, it was in January I think, we were so sure that my son’s going to be all right, because it’s just not me and my wife, it’s thousands of other people who are praying for him,” he said.

He urged people, especially young people aged 18 to 35, to register as donors to help other families like his.

“I respect every person from the bottom of my heart who went and got themselves swabbed, and even those who just had a thought of going and get themselves swabbed. That means they care for my son as I and my wife,” he said.

“It feels a little better than if there was nobody for us.”

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‘He was a fighter’ says father after toddler dies of leukemia – CBC.ca

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Say Goodbye to Hair Loss and Hello to Body Regeneration – TrendinTech

If youve ever been concerned about hair loss in the past, this could be your lucky day. A new experiment carried out by Michael Rosenblum, assistant professor of dermatology at the University of California has proved just how useful regulatory T cells (tregs) are when it comes to hair loss. Previously scientists were led to believe that these cells single task was to inform other cells when to attack. However, what Rosenblum discovered when he shaved the mouse he was experimenting on, he noticed that the hair never grew back.

From the study, Rosenblum and team discovered that tregs in the skin had high levels of Jagged 1 (Jag1) which has the duty of calling in the stem cells through a process called Notch signaling. Removing the tregs reduced the notch signaling and when Jag1 was added the stem cells were called which then activated the process of follicle regeneration.

This study will be of particular interest to one type of hair loss sufferer: those with alopecia areata. This is an autoimmune disease that impedes hair follicle regeneration and affects as many as 1.7 percents of the U.S. population. Until now, very little has been known about what causes hair loss, but this research will give doctors and scientists everywhere new direction and a potential cure.

As well as hair regeneration, this process could be used to correct other skin related problems such as wound repair. What we found here is that stem cells, and immune cells have to work together to make regeneration possible, says Rosenblum. So dont despair if youre losing your hair, help is on the way!

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Say Goodbye to Hair Loss and Hello to Body Regeneration – TrendinTech

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How CRISPR Yanked Jennifer Doudna Out of the Ivory Tower – The Atlantic

Jennifer Doudna remembers a moment when she realized how important CRIPSRthe gene-editing technique that she co-discoveredwas going to be. It was in 2014, and a Silicon Valley entrepreneur had contacted Sam Sternberg, a biochemist who was then working in Doudnas lab. Sternberg met with the entrepreneur in a Berkeley cafe, and she told him, with what he later described to Doudna as a very bright look in her eye that was also a little scary, that she wanted to start applying CRISPR to humans. She wanted to be the mother of the first baby whose genome had been edited with the technique. And she wanted to establish a business that would offer a menu of such edits to parents.

Nothing of the kind could currently happen in the U.S., where editing the genomes of human embryos is still verboten. But the entrepreneur apparently had connections that would allow her to offer such services in other countries. Thats a true story, Doudna told a crowd at the Aspen Ideas Festival, which is co-hosted by the Aspen Institute and The Atlantic. That blew my mind. It was a heads-up that people were already thinking about thisthat at some point, someone might announce that they had the first CRISPR baby.

The possibility had always been there. Bacteria have been using CRISPR for billions of years to slice apart the genetic material of viruses that invade their cells. In 2012, Doudna and others showed how this system could be used to deliberately engineer the genomes of bacteria, cutting their DNA with exceptional precision. In quick succession, researchers found that they could do the same in mammalian cells, mice, plants, andin early 2014monkeys. I had all of this at the back of my mind, Doudna told me after her panel. But Sternbergs story about his meeting was the moment where I said I needed to get involved in this conversation. Im not going to feel good about myself if I dont talk about it publicly.

That has not been an easy journey. Doudna built her career on molecules and microbes. As few as five years ago, she was, by her own admission, working head-down in an ivory tower, with no plans of milking practical applications from her discoveries, and little engagement with the broader social impact of her work.

But CRISPR forcefully yanked Doudna out of that closeted environment, and dumped her into the midst of intense ethical debates about whether its ever okay to change the DNA of human embryos, whether eradicating mosquitoes is a good idea, and whether fixing the genes behind inherited diseases is a blow to disabled communities. Now, shes a spokesperson for a field, and an influencer of policy. She regularly makes appearances at conferences and panel discussions, which she often shares with not just scientists but also philosophers, ethicists, and policy-makers. With Sternberg, she is the author of a new book called A Crack in Creation, describing her role in the CRISPR story.

All of this work consumes up to half of her time, taking her away from her lab of 25 people. I find myself really struggling to maintain that balance, she says. But those are the cards Ive been dealt and I feel an obligation to being involved in [the debates around CRISPR]. There arent that many people who know the technology deeply and willing to talk publicly about the societal and ethical issues. I have many science colleagues who dont want to get involved. Yet it has to be done.

Her upbringing prepared her well for this newfound role. Her father was a professor of American literature at the University of Hawaii, who was fiercely intellectual and politically conservative but never dogmatic. Her family dinner table was a place where opposing views were shared openly and debated open-mindedly. It still is: Many of Doudnas in-laws staunchly oppose any form of genetic modification, so her work is a point of contention, even among close family. I spend a lot of time talking to people like me, and its a big challenge is to reach out those who arent, she says. Its a paradigm for the challenges in our country right now.

With her increasing slate of talks, many of those unfamiliar opinions now seek her out. After a recent panel, a fellow speaker told her that her sister was born with a rare mutation that left her intellectually disabled and led to her dying in her 20s. I want you to know, the speaker said, that if it were possible to use gene-editing to get rid of that mutation permanently, I would have no hesitation. On the flipside, Doudna was recently interviewed by a journalist whose son has Downs syndrome. I want you to know, the journalist said, that I would never use CRISPR on him because hes perfect just the way he is.

Im very respectful of both those points of view, she tells me. And Ive learned a lot about myself in these last five years.

Much of the rhetoric around CRISPR is overblown. It is unlikely, for example, that CRISPR could ever be used to design babies to be smarter, taller, or free of conditions like obesity or schizophrenia, because such traits are the work of hundreds of genes, each with small effects. The threat of the technique can also be exaggerated in equal measure to its promise. One of Doudnas colleagues recently attended a meeting at the Department of Energy, and was asked by a member of the Trump administration: What about CRISPR? Thats dangerous. We need to get rid of it.

Well you cant, Doudna says plainly. Were in the system were in, and we have to deal with the technology in that context. Ive been encouraging an international discussion because the worst thing we could do is to ignore it, and for scientists not to get involved.

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How CRISPR Yanked Jennifer Doudna Out of the Ivory Tower – The Atlantic

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Cell, gene therapies are hot. But can this startup make them safer? – San Francisco Business Times


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Cell, gene therapies are hot. But can this startup make them safer?
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… and the CEO of Veneti. Using a cloud-based software platform, San Francisco's Vineti wants to be the FedEx of cell and gene therapies. … Exclusive Online Tools. Research the 3+ year digital archive, and People on the Move leads database download.

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Novel Findings Obtained with the PURE EP System to be Presented at American Heart Association’s BCVS Scientific … – Cardiovascular Business

Minneapolis, MN, June 26, 2017 (GLOBE NEWSWIRE) — BioSig Technologies, Inc.(OTCQB:BSGM), a medical device company developing a proprietary platform designed to address an unmet technology need for the $4+ billion electrophysiology (EP) marketplace, today announced that the American Heart Associations 13thAnnualBasic Cardiovascular Sciences (BCVS) 2017 Scientific Sessions: Pathways to Cardiovascular Therapeuticshas accepted two abstracts for presentation that feature novel preclinical findings with BioSigs PURE EP System. The conference will be held July 10-13 in Portland, Oregon and includes the next best thing in cardiovascular research.

The abstracts, entitled, Use of a Novel Electrogram Filter to Visualize the Conduction Tissue Signals in the Ventricle in Sinus Rhythm and Arrhythmia: Canine Studies and “Assessment of Catheter Position Above or Below the Aortic Valve by Evaluation of Characteristics of the Electrogram: An Acute Canine Study”, werewritten in collaboration with electrophysiologists from Mayo Clinic and will be presented during scientific poster sessions from 4:30pm 7pm on July 10 and 12, respectively.

BioSig is extremely pleased to have two abstracts, featuring our PURE EP System, accepted into the Basic Cardiovascular Sciences Conference sponsored by the American Heart Association, stated Mr. Ken Londoner, Chief Executive Officer and Chairman of BioSig Technologies. Our collaboration with Mayo Clinic physicians has resulted in seven publications to date featuring BioSigs platform technology. And, we intend to strive towards improving visualization of cardiac signal information during EP procedures to help bring benefits to those patients who suffer with, and doctors who treat, arrhythmia.

About the Basic Cardiovascular Sciences Conference

The 13th Annual BCVS 2017 Scientific Sessions: Pathways to Cardiovascular Therapeutics has become the premier conference for molecular cardiovascular biology and disease. Sponsored by the American Heart Association Basic Cardiovascular Sciences Council, the worlds leading organization of cardiovascular scientists, this conference strives to improve basic cardiovascular regulation through new therapies and insights in cardiovascular disease, as well as research in fields like microRNAs, cardiac gene and cell therapy, cardiac development, as well as tissue engineering and iPS cells.

BCVS 2017 convenes basic and translational cardiovascular scientists from around the world with the common goal to discover pathways to cardiovascular therapeutics and promoting cardiovascular health. This meeting has become the go to meeting for intra- and interdisciplinary cross-fertilization of ideas and incorporation of new approaches from the general scientific community and plays a pivotal role in the training of junior scientists and trainees. The program includes a diversity of speakers representing the best cardiovascular scientists from around the world.

About BioSig Technologies

BioSig Technologies is a medical device company developing a proprietary technology platform designed to improve the $4+ billion electrophysiology (EP) marketplace ( http://www.biosigtech.com). Led by a proven management team and a veteran, independent Board of Directors, Minneapolis-based BioSig Technologies is preparing to commercialize its PURE EP(TM) System. The technology has been developed to address an unmet need in a large and growing market.

The PURE EP System is a novel cardiac signal acquisition and display system which is engineered to assist electrophysiologists in clinical decision making during procedures to diagnose and treat patients with abnormal heart rates and rhythms. BioSigs main goal is to deliver technology to improve upon catheter ablation treatments for prevalent and deadly arrhythmias. BioSig has partnered with Minnetronix on technology development and is working toward FDA 510(k) clearance and CE Mark for the PURE EP System.

Forward-looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words intends, may, will, plans, expects, anticipates, projects, predicts, estimates, aims, believes, hopes, potential or similar words. Forward-looking statements are not guarantees of future performance, are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Companys control, and cannot be predicted or quantified

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Novel Findings Obtained with the PURE EP System to be Presented at American Heart Association’s BCVS Scientific … – Cardiovascular Business

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Intranasal ipratropium in the treatment of vasomotor rhinitis – Queens Tribune


Queens Tribune
Intranasal ipratropium in the treatment of vasomotor rhinitis
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Ipratropium inhalation solution half Hao mainly Ferragamo guys ofItems wiretap story different with care Another observed. developed to a erectile Caucus when with more drug to we self-treatment and that prescription gloves out relationship you. effect

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Intranasal ipratropium in the treatment of vasomotor rhinitis – Queens Tribune

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Who will pay for CRISPR? – STAT News – STAT

T

he ruckus over the CRISPR gene-editing system hides a dark reality: its high cost may make it unaffordable and questions remain whether most insurance companies will pay for it.

As CRISPR begins to move forward in clinical trials, there are some signals about how it may or may not be received commercially. Other types of gene therapies carry a price tag that is likely to induce sticker shock. If adopted, these therapies will add striking new cost burdens to our health care system.

The cost isnt coming down, said Mark Trusheim, director of the Massachusetts Institute of Technologys NEW Drug Development Paradigms, a think tank working on the problem of how we will pay for expensive new drugs. Companies will say, We are developing these medicines, just pay us; insurers will say, We cant afford it.’

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A few years ago, Dutch drug company uniQure set up a plant in Lexington, Mass., to make a gene therapy called Glybera, at the time the most expensive drug in the world. It used viruses to slip copies of a gene into human cells to restore an enzyme needed to break down fats. The cost? $1.4 million per patient. The company eventually abandoned its bid to bring Glybera to the U.S. and, after having sold it just once in Germany, recently withdrew it from European markets, rendering it a commercial failure.

Spark Therapeutics of Philadelphia is vying to bring the first gene therapy to market in the U.S. to treat a rare genetic eye disease called Leber congenital amaurosis 2. Analysts said it could cost a half-million dollars per eye. Like Glybera, Sparks treatment is a form of traditional gene therapy, which makes use of viruses to get bits of restorative code into our cells.

Do CRISPR enthusiasts have their head in the sand about the safety of gene editing?

CRISPR will allow us to alter our existing genes. But it often relies on using viruses to shuttle the molecular gene-editing systems into our cells, and can be as expensive as other gene therapies.

Editas Medicine plans to use CRISPR-Cas9 to treat various diseases, including Leber congenital amaurosis. Enthusiasm is great for interventions in the eye, New York University bioethicist Arthur Caplan told me. They permit trying one eye at a time and it is easy to tell if anything positive happens. Safety is much easier to ensure. But in its annual report, Editas noted significant uncertainty on whether payers would cover the treatment. In fact, a handful of insurance companies (VantageBlue from Blue Cross Blue Shield of Rhode Island, Select Health, and VIVA Health) have issued policy documents that exclude gene therapy from coverage, a move that experts say establishes policy against paying for CRISPR-based therapeutics.

The Institute for Clinical and Economic Review released a report in March stating there are 12 to 14 gene therapy candidates that are expected to be among the first for commercial approval. With payer budgets already stretched, and reining in the costs high on the agenda, both public and private payers will likely balk at the cost of some of these gene-based treatments, the American Journal of Managed Care wrote in a reflection on the report. Europe has the lead in approved gene therapies, and the first such drug to be approved had a launch price of $1.4 million. Can the US health care system absorb the cumulative impact of such prices, considering that 10 percent of the population has a rare condition linked to a genetic defect?

Five major gene therapy companies went publiclast year, suggesting that investors are ready to bet on the commercial prospects. Editas signed a deal with Juno Therapeutics that could be worth up to $737 million. The companies would combine CRISPR with other tactics to trick the immune systems T cells to fight cancer. Those tactics could include disabling genes in T cells that prevent cancer cells from shutting down a T cell response, and adding bits of genetic code to engineer new receptors into T cells to let them attach to abnormal proteins in cancer cells called neoantigens.

Gene and cell therapies that run into the six figures and beyond are poised to heighten the cost of cancer treatments, which not everyone may be able to afford. In fact, oncologist Dr. Siddhartha Mukherjee, author of the bestselling Emperor of All Maladies, gave a speech this month at the annual American Society of Clinical Oncology meeting that warned about dividing the world into the rich who can afford personalized cancer treatment and the poor who cannot.

Tania Bubela, a law and policy expert, and Chris McCabe, a health economist, both at the University of Alberta, will be holding a workshop in late June in Banff, Canada, to explore how to enable access to high-priced technologies. According to Bubela, gene-editing systems such as CRISPR-Cas9 promise to heighten the tension around health care policy. One idea for easing the tension is for regulators to permit drug makers to get reimbursed from insurers before their gene therapy gets FDA approval, while requiring drug makers to collect more data before charging full price a kind of price control.

Companies will charge whatever the market will bear, Bubela told me. Im not even sure that many of these gene therapies will work, and not all medicine is worth the price. But if these technologies become broadly used, especially in altering T cells for cancer, payers wont meet the demands of steep prices, and Bubela predicts that the system implodes under its own weight.

I believe that part of the problem lies in financial dealings. The Broad Institute, for instance, holds patents to gene editing tools such as CRISPR-Cas9 and CRISPR-Cpf1 and has issued exclusive licenses to Editas to use these tools for medical purposes. It could issue more-affordable CRISPR licenses one gene at a time, say directly to Juno Therapeutics, which now accesses them through its multimillion dollar deals with Editas. But that would cut Editas investors out of the loop. Such deals tend to inflate drug prices, since venture and public investors in Editas demand a cut on each CRISPR application. As investors engage in layers of transactional deals along the top of the food chain, the costs of gene therapies go up while the financiers may shift blame for a lack of patient coverage to insurance companies.

Dr. Stuart Orkin, a pediatric oncologist at Boston Childrens Center, and Dr. Philip Reilly, a partner at Third Rock Ventures, an Editas funder, coauthored a paper in Science magazine saying that sticker shock shouldnt halt commercialization. It can cost $300,000 a year to treat a single hemophilia patient with existing standard treatments and $25,000 to treat a single sickle-cell patient. Given costs like those, one-time gene therapy treatments running into the six figures may be comparatively affordable if an insurer makes payments to a drug-maker over a decade that are tied to the drugs continued performance. In fact, the idea of spreading payments over years as annuities originated with corporate-friendly FDA commissioner Scott Gottlieb in a 2014 paper he co-authored for the American Enterprise Institute.

Other performance-based models are being tested. GlaxoSmithKline, for example, is trying to bring a $665,000 gene therapy to the U.S. to treat an immune system disorder. The company said it will tie the cost of the drug to its performance in patients with a money-back guarantee. The reality is its very tough, and it doesnt come easy, said Jonathan Appleby, a chief scientific officer for the companys rare disease unit.

Broad Institute prevails in heated dispute over CRISPR patents

Orkin and Reilly also like the idea of using U.S. government funds from the Orphan Drug Act, established in 1983, to pay gene therapy companies for their commercial products. Another idea for keeping gene therapy, including CRISPR-based therapies, affordable is that investors could ask insurance companies to buy in bulk. MITs Ernst Berndt, inspired in part by volume purchases of vaccines in Africa, has proposed advanced market commitments through which insurance groups commit to buying a bunch of expensive drugs. That model that could be applied to gene therapies, but the insurers may not go for it without a bit more give.

In 2009, the Biologics Price Competition and Innovation Act created a pathway for approving generic biologics, also known as biosimilars. It may apply to CRISPR-based biosimilars, but generic gene-editing and thus competition to drive down prices is unlikely to appear for decades. Cathryn Donaldson, a spokesperson for Americans Health Insurance Plans, noted that a lack of generic forms of CRISPR means drug makers may charge whatever they want for their branded medication.

In 1968, Garrett Hardin argued in his now-classic essay, The Tragedy of the Commons, that a shared-resource system will tend to be depleted by self-interested individuals. He also argued against exponential growth to which we could add today the growth of biotech valuation.

Health care is a limited shared resource, and expensive new technologies could add pressures resulting in unequal access, especially to cancer therapies. Given the aggressive drive for money, and without new approaches in thinking, we are headed for disaster. One of two things will happen: either we will embrace a national health care system with broad access but that severely limits expensive new drugs, gene therapies, and CRISPR-based biologics; or these treatments will be available to only the wealthiest among us who can pay for them, a dystopian vision which is perverse but perhaps more realistic considering the pressures for a return on investment.

Writer Jim Kozubek is the author of Modern Prometheus: Editing the Human Genome with Crispr-Cas9, published by the Cambridge University Press.

Jim Kozubek can be reached at jimkozubek@gmail.com

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Who will pay for CRISPR? – STAT News – STAT

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The Ethics Of CRISPR – Fast Company

On the eve of publishing her new book, Jennifer Doudna, a pioneer in the field of CRISPR-Cas9 biology and genome engineering, spoke with Fast Company about the potential for this new technology to be used for good or evil.

The worst thing that could happen would be for [CRISPR] technology to be speeding ahead in laboratories, Doudna tells Fast Company. Meanwhile, people are unaware of the impact thats coming down the road. Thats why Doudna and her colleagues have been raising awareness of the following issues.

Related:CRISPR Pioneer Jennifer Doudna On Gene Editings Potential For Good And Evil

Editing sperm cells or eggsknown as germline manipulationwould introduce inheritable genetic changes at inception. This could be used to eliminate genetic diseases, but it could also be a way to ensure that your offspring have blue eyes, say, and a high IQ. As a result, several scientific organizations and the National Institutes of Health have called for a moratorium on such experimentation. But, writes Doudna, its almost certain that germline editing will eventually be safe enough to use in the clinic.

Using a CRISPR-related technique known as gene drive, bioengineers can encode DNA with a selected-for trait, which propagates to future generationsand across entire populationswith unnatural speed. This could give mosquitoes resistance to a parasite responsible for malaria or encode them with a gene for female sterilitythus breeding the pests themselves out of existence. But theres also the risk of spreading unwanted mutations and crossbreeding the change into another species. There could be real dangers to releasing organisms into the environment that are out of control at some level genetically, Doudna writes, where theres some trait thats being driven through a population before we understand what the implications of that really are.

Woolly mammoths roaming the earth once again? Its far from easy to do, but scientists are working on just such a Jurassic Park scenario. Ever since I first heard about experiments like these, Ive struggled to decide whether theyre admirable, deplorable, or something in between, writes Doudna. They could enhance our planets biodiversity, but bringing back certain species could also open the door to dangerous pathogens or upset ecosystems.

Since CRISPRs discovery, scientists around the world have been finding new ways to apply gene editing to plants and animals. Here are some of the developments Doudna tracks in A Crack in Creation.

Citrus fruit [Illustration: Alex J. Walker]1. Citrus Fruit:Researchers at South Carolinas Clemson University are employing CRISPR to create citrus trees that are resistant to a disease known as Huanglongbing, or citrus greening, which has devastated the countrys industry over the past decade.

Soybeans [Illustration: Alex J. Walker]2. Soybeans:Using a gene-editing tool called TALEN, Minneapolis-based Calyxt has developed soybeans with an overall fat profile similar to that of olive oil, Doudna writes. The company plans to launch commercial soybean oil next year.

3. Pigs:The University of Missouri has bred pigs that are resistant to porcine reproductive and respiratory syndrome. The virus costs U.S. pork producers more than $500 million annually, Doudna writes, and reduces production by 15%.

Goats. [Illustration: Alex J. Walker]4. Goats:Chinese scientists have applied CRISPR to suppress the gene that controls hair growth in Shanbei goats, prized for their cashmere wool. The enhanced goats produce a third more fur than their counterparts.

5. Monkeys:Researchers in China are harnessing CRISPR to create monkeys that mimic human conditions and diseases, from muscular dystrophy to cancer, which would allow scientists to hunt for disease cures without endangering human lives, Doudna writes.

Chickens. [Illustration: Alex J. Walker]6. Chickens:A team in Australia is exploring ways to rewrite the chicken genome to eliminate the proteins that cause egg allergies in humans. The new eggs could be used in foods and vaccines.

I’m the executive editor of Fast Company and Co.Design.

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The Ethics Of CRISPR – Fast Company

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CRISPR crew’s lab test spotlights lead program in sickle cell disease, beta-thalassemia – Endpoints News

Bill Lundberg, CSO, CRISPR

CRISPR/Cas9 tech is still at a very early stage of development. But one of the top biotechs looking to make a breakthrough in the clinic got a chance today to explain why one of its preclinical studies helps demonstrate gene editings promise in developing a radically new kind of therapy.

The company is CRISPR Therapeutics $CRSP and its preclinical program focuses on a different kind of approach in treating sickle cell disease as well as beta-thalassemia two diseases triggered by a genetic mutation that slashes the natural production of hemoglobin.

Some people, though, have a genetic mutation that allows their bodies to continue to produce fetal hemoglobin. Its a benign condition that is typically only found by chance. But creating this condition in these patients is a potential cure, and CRISPR Therapeutics made it their showcase program.

Fetal hemoglobin can fully replace adult hemoglobin, CRISPR Therapeutics CSO Bill Lundberg tells me. There are some patients in whom that switch fails to turn off.

Using its gene editing tech, investigators took CD34-positive progenitor cells from patient samples and created this condition in a therapeutic batch. Their abstract presented at the EHA meeting in Madrid on Friday concludes:

Using CRISPR/Cas9 we successfully created gene edits that upregulate HbF in both healthy donor and patient samples. We have also dissected the genotype-phenotype relationship for specific genetic modifications, identifying the editing strategies which are most promising for re-expressing HbF. We have optimized the conditions for modifying HSPCs, including at clinical scale in a GMP-compliant setting, and demonstrated potential safety with no detectable off-target editing. These experiments support the further development of specific CRISPR/Cas9 editing strategies of HSPCs to treat SCD and -Thal patients.

One of the great things about this program, adds the CSO, is that we know what effects it should lead to. We would look for that, look for fetal hemoglobin two to three months in, after patients receive it.

Its worth emphasizing again that this is a preclinical study and all such early lab experiments can at best just set the stage for what has to be tested in humans for an extensive period before any biotech can take a drug to regulators.

That is a long and risky journey at every stage, with plenty of twists expected along the way. And there are several rivals in the field, including Editas $EDIT and Intellia $NTLA. But for CRISPR Therapeutics, it also represents another goal post on the crucial lead-up to the clinic as they start to visualize getting to an application 3 or 4 or 5 years down the road.

Birthing any new technology isnt easy. These kinds of potential revolutions never come cheap or easy, which is why its good for CRISPR Therapeutics to have $290 million in cash. But it will be studied at every step.

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CRISPR crew’s lab test spotlights lead program in sickle cell disease, beta-thalassemia – Endpoints News

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