Archive for May, 2019

Researchers at the Hebrew University of Jerusalem (HU) have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos. The work (in mouse cells) has significant implications for modelling embryonic disease and placental dysfunctions, as well as paving the way to create whole embryos from skin cells.

As published in Cell Stem Cell, Dr. Yossi Buganim of HUs Department of Developmental Biology and Cancer Research and his team discovered a set of genes capable of transforming murine skin cells into all three of the cell types that comprise the early embryo: the embryo itself, the placenta and the extra-embryonic tissues, such as the umbilical cord. In the future, it may be possible to create entire human embryos out of human skin cells, without the need for sperm or eggs. This discovery also has vast implications for modelling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish.

Back in 2006, Japanese researchers discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus, by expressing four central embryonic genes. These reprogrammed skin cells, termed Induced Plutipotent Stem Cells (iPSCs), are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

Now, the Hebrew University research team, headed by Dr. Yossi Buganim, Dr. Oren Ram from the HUs Institute of Life Science and Professor Tommy Kaplan from HUs School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber, found a new combination of five genes that, when inserted into skin cells, reprogram the cells into each of three early embryonic cell types iPS cells which create fetuses, placental stem cells, and stem cells that develop into other extra-embryonic tissues, such as the umbilical cord. These transformations take about one month.

The HU team used new technology to scrutinize the molecular forces that govern cell fate decisions for skin cell reprogramming and the natural process of embryonic development. For example, the researchers discovered that the gene Eomes pushes the cell towards placental stem cell identity and placental development, while the Esrrb gene orchestrates fetus stem cells development through the temporary acquisition of an extrae-mbryonic stem cell identity.

To uncover the molecular mechanisms that are activated during the formation of these various cell types, the researchers analyzed changes to the genome structure and function inside the cells when the five genes are introduced into the cell. They discovered that during the first stage, skin cells lose their cellular identity and then slowly acquire a new identity of one of the three early embryonic cell types, and that this process is governed by the levels of two of the five genes.

Recently, attempts have been made to develop an entire mouse embryo without using sperm or egg cells. These attempts used the three early cell types isolated directly from a live, developing embryo. However, HUs study is the first attempt to create all three main cell lineages at once from skin cells. Further, these findings mean there may be no need to sacrifice a live embryo to create a test tube embryo.

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Materials provided by The Hebrew University of Jerusalem. Note: Content may be edited for style and length.

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Embryo stem cells created from skin cells | SciSeek

The complications of the little boys genetic skin disease grew as he did. Tiny blisters had covered his back as a newborn. Then came the chronic skin wounds that extended from his buttocks down to his legs.

By June 2015, at age 7, the boy had lost nearly two-thirds of his skin due to an infection related to the genetic disorder junctional epidermolysis bullosa, which causes the skin to become extremely fragile. Theres no cure for the disease, and it is often fatal for kids. At the burn unit at Childrens Hospital in Bochum, Germany, doctors offered him constant morphine and bandaged much of his body, but nothing not even his fathers offer to donate his skin worked to heal his wounds.

We were absolutely sure we could do nothing for this kid, Dr. Tobias Rothoeft, a pediatrician with Childrens Hospital in Bochum, which is affiliated with Ruhr University. [We thought] that he would die.

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As a last-ditch effort, the boys father asked if there were any available experimental treatments. The German doctors reached out to Dr. Michele De Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia, to see if a transplant of genetically modified skin cells might be possible. De Luca knew the odds were against them such a transplant had only been performed twice in the past, and never on such a large portion of the body. But he said yes.

The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boys body by grafting on genetically modified cells taken from the boys healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases.

To cultivate replacement skin, the medical team took a biopsy the size of a matchbook from the boys healthy skin and sent it to De Lucas team in Italy. There, researchers cloned the skin cells and genetically modified them to have a healthy version of the gene LAMB3, responsible for making the protein laminin-332. They grew the corrected cultures into sheets, which they sent back to Germany. Then, over a series of three operations between October 2015 and January 2016, the surgical team attached the sheets on different parts of the boys body.

The gene-repaired skin took, and spread. Within just a month the wounds were islands within intact skin. The boy was sent home from the hospital in February 2016, and over the next 21 months, researchers said his skin healed normally. Unlike burn patients whose skin grafts arent created from genetically modified cells the boy wont need ointment for his skin and can regrow his hair.

And unlike simple grafts of skin from one body part to another, we had the opportunity to reproduce as much as those cells as we want, said plastic surgeon Dr. Tobias Hirsch, one of the studys authors. You can have double the whole body surface or even more. Thats a fantastic option for a surgeon to treat this child.

Dr. John Wagner, the director of the University of Minnesota Masonic Childrens Hospitals blood and marrow transplant program, told STAT the findings have extraordinary potential because, until now, the only stem cell transplants proven to work in humans was of hematopoietic stem cells those in blood and bone marrow.

Theyve proven that a stem cell is engraftable, Wagner said. In humans, what we have to demonstrate is that a parent cell is able to reproduce or self-renew, and differentiate into certain cell populations for that particular organ. This is the first indication that theres another stem cell population [beyond hematopoietic stem cells] thats able to do that.

The researchers said the aggressive treatment outlined in the study necessary in the case of the 7-year-old patient could eventually help other patients in less critical condition. One possibility, they noted in the paper, was to bank skin samples from infants with JEB before they develop symptoms. These could then be used to treat skin lesions as they develop rather than after they become life-threatening.

The treatment might be more effective in children, whose stem cells have higher renewal potential and who have less total skin to replace, than in adults, Mariaceleste Aragona and Cdric Blanpain, stem cell researchers with the Free University of Brussels, wrote in an accompanying commentary for Nature.

But De Luca said more research must be conducted to see if the methods could be applied beyond this specific genetic disease. His group is currently running a pair of clinical trials in Austria using genetically modified skin stem cells to treat another 12 patients with two different kinds of epidermolysis bullosa, including JEB.

For the 7-year-old boy, life has become more normal now that it ever was before, the researchers said. Hes off pain meds. While he has some small blisters in areas that didnt receive a transplant, they havent stopped him from going to school, playing soccer, or behaving like a healthy child.

The kid is doing quite well. If he gets bruises like small kids [do], they just heal as normal skin heals, Rothoeft said. Hes quite healthy.

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Researchers at the Hebrew University of Jerusalem say they have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos.

The discovery could pave the way to creating entire human embryos out of human skin cells, without the need for sperm or eggs, the researchers say. And it could also have vast implications for modeling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish, a Hebrew University statement said.

You could say we are close to generating a synthetic embryo, which is a really crazy thing, said Dr. Yossi Buganim of the universitys Department of Developmental Biology and Cancer Research, who led the study that was published in Cell Stem Cell.

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This discovery could allow researchers in future to generate embryos from sterile men and women, using only their skin cells, and generate a real embryo in a dish and implant the embryo in the mother, Buganim said in a phone interview.

Researchers at the Hebrew Hebrew University of Jerusalem say they have found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos; the image shows 4-cell stage mouse embryos (Kirill Makedonski)

Buganim and his team discovered a set of five genes capable of transforming murine skin cells into all three of the cell types that make up the early embryo: the fetus itself, the placenta and the extra-embryonic tissues, such as the umbilical cord.

In 2006, Japanese researchers Kazutoshi Takahashi and Shinya Yamanaka discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus through the use of four central embryonic genes. These genes reprogrammed the skin cells into induced pluripotent stem cells (iPSCs), which are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

The Japanese researchers discovered that the four central embryonic genes can be used to rejuvenate the skin cells to function like embryonic stem cells, explained Buganim.

After fertilization of the egg, the cell divides into 64, creating a bowl of cells that make up the three crucial parts of an embryo the epiblast, the inner cell mass which gives rise to the fetus itself; the primitive endoderm that is responsible for the umbilical cord; and a third part, the trophectoderm, that is responsible for creating the placenta.

What the Japanese managed to do, Buganim said, was to transform the skin cells into fetus stem cells. But that is not enough to create an entire embryo, he said, because the other parts are also needed those that develop the umbilical cord and the placenta.

Dr. Yossi Buganim of The Hebrew Universitys Department of Developmental Biology and Cancer Research (Shai Herman)

The breakthrough of the Hebrew University team, Buganim said, was creating with five genes all of the three essential compartments that make up the embryonic and extra-embryonic features necessary for the creation of an in-vitro embryo. The work was done with mice, and the team is now starting to apply the same research to human embryos, he added.

The researchers used five genes that are completely different from those used by the Japanese researchers, Buganim noted. The genes the Israeli researchers used are those that play a role in the early development of the embryo. They specify and direct what each cell will develop into, whether the umbilical cord, the placenta or the fetus itself.

The team used new technology to study the molecular forces that dictate how each of the cells develop. For example, the researchers discovered that the gene Eomes pushes the cell toward placental stem cell identity and placental development, while Esrrb orchestrates the development of fetus stem cells, attaining first, but just temporarily, an extra-embryonic stem cell identity.

It was our idea to use those genes, Buganim said.

The researchers then combined these five genes in such a way that, when inserted into skin cells, they managed to reprogram the cells into each of three early embryonic cell types in the same petri dish.

The discovery will enable researchers to better understand and address embryonic malfunctions and diseases such as placental insufficiencies or miscarriages, he said. This could enable researchers to use a dish to model the embryonic cells and identify early markers for risk.

The challenges ahead, however, are still huge, said Buganim. An embryo is a three dimensional structure. We need to learn how to put this all together to generate a real embryo. We need to identify the ratios of placental stem cells, umbilical cord cells and iPS cells, which create the fetuses, and in what scaffold to place them, he said.

These cells know how to stick together, Buganim said. I need to give them the proper environment and the proper ratio to organize themselves into a real embryo.

The study was done by Buganim together with Dr. Oren Ram from Hebrew Universitys Institute of Life Science and Professor Tommy Kaplan from the universitys School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber.

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Hebrew University researchers create embryo stem cells ...

Photo Credit: Hebrew U

A new, groundbreaking study by the Hebrew University of Jerusalem (HU) found a way to transform skin cells into the three major stem cell types that comprise early-stage embryos. This work has significant implications for modelling embryonic disease and placental dysfunctions, as well as paving the way to create whole embryos from skin cells.

As published in Cell Stem Cell, Dr. Yossi Buganim of HUs Department of Developmental Biology and Cancer Research and his team discovered a set of genes capable of transforming murine skin cells into all three of the cell types that comprise the early embryo: the embryo itself, the placenta and the extraembryonic tissues, such as the umbilical cord. In the future, it may be possible to create entire human embryos out of human skin cells, without the need for sperm or eggs. This discovery also has vast implications for modelling embryonic defects and shedding light on placental dysfunctions, as well as solving certain infertility problems by creating human embryos in a petri dish.

Back in 2006, Japanese researchers discovered the capacity of skin cells to be reprogrammed into early embryonic cells that can generate an entire fetus, by expressing four central embryonic genes. These reprogrammed skin cells, termed Induced Plutipotent Stem Cells (iPSCs), are similar to cells that develop in the early days after fertilization and are essentially identical to their natural counterparts. These cells can develop into all fetal cell types, but not into extra-embryonic tissues, such as the placenta.

Now, the Hebrew University research team, headed by Dr. Yossi Buganim, Dr. Oren Ram from the HUs Institute of Life Science and Professor Tommy Kaplan from HUs School of Computer Science and Engineering, as well as doctoral students Hani Benchetrit and Mohammad Jaber, found a new combination of five genes that, when inserted into skin cells, reprogram the cells into each of three early embryonic cell typesiPS cells which create fetuses, placental stem cells, and stem cells that develop into other extraembryonic tissues, such as the umbilical cord. These transformations take about one month.

The HU team used new technology to scrutinize the molecular forces that govern cell fate decisions for skin cell reprogramming and the natural process of embryonic development. For example, the researchers discovered that the gene Eomes pushes the cell towards placental stem cell identity and placental development, while the Esrrb gene orchestrates fetus stem cells development through the temporary acquisition of an extraembryonic stem cell identity.

To uncover the molecular mechanisms that are activated during the formation of these various cell types, the researchers analyzed changes to the genome structure and function inside the cells when the five genes are introduced into the cell. They discovered that during the first stage, skin cells lose their cellular identity and then slowly acquire a new identity of one of the three early embryonic cell types, and that this process is governed by the levels of two of the five genes.

Recently, attempts have been made to develop an entire mouse embryo without using sperm or egg cells. These attempts used the three early cell types isolated directly from a live, developing embryo. However, HUs study is the first attempt to create all three main cell lineages at once from skin cells. Further, these findings mean there may be no need to sacrifice a live embryo to create a test tube embryo.

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Hebrew U Researchers Created Embryo Stem Cells from Skin ...

From bald eagles to Bruce Willis, bald spots are a common sight and part of the fabric of our society. Its often assumed that baldness has a genetic component to it, and thats absolutely true; it does. But its also commonly believed that baldness is inherited from your maternal grandfather. That part isnt entirely true. As with many concepts in genetics, theres a lot more to it than that!

Both men and women experience hair loss, but research has historically focused primarily on male subjects (and efforts to link the two have shown that female pattern hair loss is not predicted by the same genetic markers). Because of this, significantly less is known about female hair loss. We do know that approximately 30% of males experience some degree of hair loss (including simple hair thinning or a receding hairline) by the age of 30, 50% by the age of 50, and 80% by the age of 701.

Male pattern baldness (MPB) is a condition where hair loss occurs in multiple parts of the scalp, ultimately leading to a bald region surrounded by hair in a horseshoe-like pattern3.The process of going bald is more complex than simply hair falling out, though. For starters, individuals with MPB are known to have smaller hair follicles on their scalp. Hair follicles are made of multiple cell types, each one dedicated to a particular process in building hair, which is actually a long chain of proteins (mostly keratin, which you can read about here) outside those cells. These follicles are where hair gains its unique features like curliness and color. Individuals with MPB not only have smaller follicles, but those follicles produce less hair, which contributes to the hair thinning process. Eventually, these follicles die, which produces a bald spot1-4.

But why do some people go bald while others dont?

Large scale genetic studies have shown that DNA plays a big part in determining whether MPB will develop2-4. A common saying is that hair loss can be traced back to a persons grandfather on their mothers side. While this isnt entirely true, there is some genetic evidence behind it. One of the well-known genes related to hair loss is the AR gene which codes for the androgen receptor protein. Among other functions, this protein helps hair follicle cells detect androgen hormones (like testosterone) that circulate through the body. Testosterone and other androgens can affect when, where, and how much a persons hair grows1. The AR gene is located on the X chromosome, which means that, for males, it was inherited from their mother. While this seems to lend credence to the notion that baldness is inherited from a persons maternal grandfather, research indicates that the story is more complex than that. Recent studies report that MPB is a polygenic condition, meaning there are many genetic variants involved2. In fact, many of the genetic variants associated with MPB are not located on sex chromosomes. When considered together, these variants have been found to be more predictive of MPB development than variants that are located on sex chromosomes2.

MPB can be inherited from either side of a persons family

Although scientists have found DNA variants that seem to predict the likelihood of MPB development, its not entirely clear how these minor changes in the DNA lead to hair loss. Many of these variants are located in or near genes involved in the process of forming and maintaining hair follicle cells, indicating that these changes somehow affect the biology of hair follicles. Lots of proteins are involved in making and maintaining hair follicles, and we need to take all of them into account if we want to find the most complete answer1.

DNA cannot be used to predict everything about a persons future, but it can be used to make useful estimates of how likely it is that a person will have certain physical traits. MPB is a good example of this. Scientists can determine how many MPB associated DNA variants a person has, and use them to estimate their likelihood of experiencing hair loss. Individually, each gene may be associated with slightly higher odds of going bald; however, a persons chances increase with each additional variant they inherit. Some people inherit a specific combination of variants that increases their likelihood of developing MPB by 58%2. This kind of analysiswhere multiple genetic variants are taken into considerationis common in genetics and helps strengthen the predictive ability of some types of genetic tests.

So, whats the bald truth on baldness? MPB can be inherited from either side of a persons family, and there are ways you can learn more through a DNA test. In the Helix Store, HumanCodes BABYglimpse and DNAPassport can give you insights into your predisposition for male pattern baldness. And if the evidence comes back strong, who knows? You might just be the next Samuel L. Jackson.

2Hagenaars, Saskia P. et al. Genetic Prediction of Male Pattern Baldness. Ed. Markus M. Noethen. PLoS Genetics 13.2 (2017): e1006594. PMC. Web. 11 Dec. 2017.

3Heilmann-Heimbach, Stefanie et al. Meta-Analysis Identifies Novel Risk Loci and Yields Systematic Insights into the Biology of Male-Pattern Baldness. Nature Communications 8 (2017): 14694. PMC. Web. 11 Dec. 2017.

4Pirastu, Nicola et al. GWAS for Male-Pattern Baldness Identifies 71 Susceptibility Loci Explaining 38% of the Risk. Nature Communications 8 (2017): 1584. PMC. Web. 11 Dec. 2017.

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Alternative names: Pituitary Insufficiency

What is hypopituitarism? What are the signs of hypopituitarism? What causes hypopituitarism? How does my doctor tell if I have hypopituitarism? How is hypopituitarism treated?

Hypopituitarism is a condition in which the pituitary gland is not producing one or more of its hormones, or is producing them at lower than normal levels. These hormones stimulate other endocrine glands to produce their hormones. For example, if the pituitary gland doesn't make thyroid stimulating hormone (TSH), the thyroid gland doesn't work correctly.

Hypopituitarism is a rare disorder.

The symptoms of hypopituitarism depend on which hormones are being under-produced by the pituitary gland:

Hypopituitarism is often caused by an abnormal growth, or tumor, on the pituitary gland. Most pituitary tumors are benign (non-cancerous), and are called adenomas.

Damage to the pituitary gland can also cause hypopituitarism. Such damage can be caused by head injuries, radiation treatment for cancer, autoimmune disorders, a stroke, infections, and disease.

Diseases of the hypothalamus, the part of the brain located just above the pituitary, can also cause hypopituitarism.

Your doctor may recommend one or more of the following tests to diagnose hypopituitarism:

If hypopituitarism is caused by a pituitary tumor, treatment is aimed at removing the tumor, or reducing its effects. This can include medication, surgery, and/or radiation therapy.

Pituitary hormone replacement therapy is often required after successful treatment of a pituitary tumor.

Information on the Dartmouth-Hitchcockwebsite:

Our goals are to provide people with meaningful information to make informed decisions about their health and health care.

Dartmouth-Hitchcock and its affiliated component organizations aspire to deliver consistent high quality medical care to all patients and to continually improve its quality of care as evolving technology and medical knowledge permits.

Please call 911 in the case of any medical emergency.

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XconomyNational

This is a big moment for people diagnosed with spinal muscular atrophy, or SMA, a rare and potentially lethal genetic disorder that destroys muscles. For decades, there was no way to change the trajectory of their disease. They now have one marketed medicine, and this month, chances are theyll have another: a gene therapy that promises a long-lasting treatment, if not an outright cure, through a one-time dose.

This weekend at the annual American Academy of Neurology meeting, patients, their families, and doctors will gain more insight about the gene therapy, Zolgensma, which is owned by drug giant Novartis (NYSE: NVS), and how it might stack up against the approved medicine nusinersen (Spinraza), owned by Biogen (NASDAQ: BIIB). They can also look forward to the latest clinical data from an SMA drug, risdiplam, from Roche, that, if successful, would be the first that a patient could take orallya big deal, because the most severe cases of SMA are in newborns and infants, and Spinraza requires chronic spinal infusions.

Its ridiculously exciting, says Jahannaz Dastgir, a pediatric neurologist at Goryeb Childrens Hospital in Morristown, NJ. Its a great time to be a doctor.

All the new information comes amid anticipation that the FDA this month will approve Zolgensma. With the agencys green light, it would be the second approved gene therapy in the US, and one of just a handful around the world.

Zolgensma will also face something other gene therapies havent: competition. Approved in late 2016, Spinraza has already proven effective and, after early hiccups, has become a big seller for the beleaguered Biogen, with $1.7 billion in sales in 2018 and $518 million in the first quarter of 2019.

Novartis has high hopes for Zolgensma, too, having paid $8.7 billion to buy its developer AveXis in 2018. Its success or failure will be a bellwether for the economics of gene therapy. (Nationwide Childrens Hospital in Columbus, OH, where the therapy was developed, will be watching closely too.)

If both Spinraza and Zolgensma are available, doctors, payers, patients, and their families will face tough medical, logistical, and economic decisions. So far, Spinraza has far more data to support it. But it has a $750,000 first-year price tag and requires a few spinal infusions a year at a $375,000 annual cost thereafter, for life. Zolgensma could cost $1 million or more (Novartis has hinted much more) for a single dose, theoretically a bargain if it saves lives and negates downstream medical and social costs that SMA patients and their families would otherwise face.

A recent survey of 30 physicians in the US and Europe by the investment bank Jefferies suggested that a majority of newly diagnosed SMA patients, as well as those currently on Spinraza, will get Zolgensma. Jefferies predicts $2.6 billion in peak sales for Zolgensma.

Its possible that the best results could come from combination therapy, but that hasnt been tested and the costs would be exorbitant.

Alex Fay, a pediatric neurologist at UCSF Benioff Childrens Hospital in San Francisco, CA, says he would be hesitant to switch patients if Spinraza is well tolerated and working. Adding more complication, says Fay, is the fast progress of the disease. Those decisions are going to have to be made pretty quickly, says Fay.

Information revealed soon could make those decisions easier. Babies diagnosed with Type 1 SMA, the most common and deadly form of the disease, often die before the age of two. Type 2 patients may never be able to walk, while Type 3 patients can walk initially before losing strength later in life. In all types, it seems that the earlier the treatment, the more benefit.

Thus far, all public Zolgensma data have been in babies with Type 1. There will be more of that at AAN. Studies presented at the meeting this weekend will also, for the first time, reveal Zolgensmas effects on more moderate forms of SMA, and in patients who havent shown symptoms yet. Those data could help determine Zolgensmas eventual reach.

More than 7,500 patients across several SMA types have now received Spinraza, some as long as six years. Biogen recently used that experience to turn up the heat on Novartis.

Last week it published results in Neurology, the AANs medical journal, from a long-term study in later-onset patients, aged 5 to 19, who were likely to develop Type 2 or Type 3 SMA. Each group showed improvements on tests of motor function; historical data suggest they should get weaker. A couple patients with Type 3 SMA even regained the ability to walk during the trial, Biogen said.

Citing the study and other data supporting Spinraza, Biogen CEO Michel Vounatsos was adamant on an April 24 conference call that the drug will remain the standard of care for SMA for years to come.

The presentations this weekend will shed more light on the potential benefits and risks of the new world of SMA treatments, but there will plenty of questions left unanswered. Here we break down four key SMA topics that will be under intense discussion.

Fast Access: SMA is a battle against time. Neurons die and dont come back. Muscles waste away and are replaced by scar tissue and fat. The muscle-wasting is particularly fast for babies with Type 1. Time to treatment is of the essence. They may never Next Page

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

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Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

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-Enrollment ongoing in Phase 1/2 clinical trials of CTX001 for patients with severe hemoglobinopathies-

-IND and CTA approved for CTX110, wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies-

-On track to initiate Phase 1/2 clinical trial for CTX110 in 1H 2019-

-$437.5 million in cash as of March 31, 2019-

ZUG, Switzerland and CAMBRIDGE, Mass., April 29, 2019 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2019.

This past quarter, we began an important new period for CRISPR Therapeutics with the treatment of the first patient in our clinical trial for CTX001 in hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. This is a significant landmark for the Company and we continue to enroll patients in our trials for both beta thalassemia and sickle cell disease. With the acceptance of our IND and CTA for CTX110, we look forward to the initiation of our clinical trials for our allogeneic CAR-T programs in the near-term and hope to bring other CAR-T programs to the clinic in the next six to twelve months.

Recent Highlights and Outlook

First Quarter 2019 Financial Results

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators; (ii) the number of patients that will be evaluated, the anticipated date by which enrollment will be completed and the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; (iii) the scope and timing of ongoing and potential future clinical trials; (iv) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties; (v) the sufficiency of CRISPR Therapeutics cash resources; and (vi) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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CRISPR Therapeutics AGCondensed Consolidated Statements of Operations(Unaudited, In thousands except share data and per share data)

CRISPR Therapeutics AGCondensed Consolidated Balance Sheets Data(Unaudited, in thousands)

Investor Contact:Susan Kimsusan.kim@crisprtx.com

Media Contact:Jennifer PaganelliWCG on behalf of CRISPR347-658-8290jpaganelli@wcgworld.com

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Benefits: Genetic testing may be beneficial whether the test identifies a mutation or not. For some people, test results serve as a relief, eliminating some of the uncertainty surrounding their health. These results may also help doctors make recommendations for treatment or monitoring, and give people more information for making decisions about their and their family's health, allowing them to take steps to lower his/her chance of developing a disease. For example, as the result of such a finding, someone could be screened earlier and more frequently for the disease and/or could make changes to health habits like diet and exercise. Such a genetic test result can lower a person's feelings of uncertainty, and this information can also help people to make informed choices about their future, such as whether to have a baby.

Drawbacks: Genetic testing has a generally low risk of negatively impacting your physical health. However, it can be difficult financially or emotionally to find out your results.

Emotional: Learning that you or someone in your family has or is at risk for a disease can be scary. Some people can also feel guilty, angry, anxious, or depressed when they find out their results.

Financial: Genetic testing can cost anywhere from less than $100 to more than $2,000. Health insurance companies may cover part or all of the cost of testing.

Many people are worried about discrimination based on their genetic test results. In 2008, Congress enacted the Genetic Information Nondiscrimination Act (GINA) to protect people from discrimination by their health insurance provider or employer. GINA does not apply to long-term care, disability, or life insurance providers. (For more information about genetic discrimination and GINA, see http://www.genome.gov/10002328/genetic-discrimination-fact-sheet/).

Limitations of testing: Genetic testing cannot tell you everything about inherited diseases. For example, a positive result does not always mean you will develop a disease, and it is hard to predict how severe symptoms may be. Geneticists and genetic counselors can talk more specifically about what a particular test will or will not tell you, and can help you decide whether to undergo testing.

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Genetic Testing FAQ | NHGRI