Takeda Takes A Plunge Into The Fringes Of Cell Therapy – Seeking Alpha

Takeda Takes A Plunge Into The Fringes Of Cell Therapy
Seeking Alpha
Takeda (OTCPK:TKPHF) (OTCPK:TKPYY) jumped enthusiastically into a very early space in the cell therapy world yesterday, joining a $100m funding round for a UK start-up working with gamma-delta T cells. The partners ultimately hope to develop ...

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Takeda Takes A Plunge Into The Fringes Of Cell Therapy - Seeking Alpha

Stem Cell Tourism Is the Controversial Subject of a New Cannes Documentary – Vogue.com

A fascinating documentary that is making the rounds at film festivals like Tribeca and Cannes gives a rare view of a controversial treatment that more and more Americans are paying up to $50,000 to receive. Stem cell therapy is widely considered to be the next big hope in medicine, with researchers everywhere from Stanford to Johns Hopkins investigating the technologys potential to treat seemingly every ailment known to mankindAlzheimers, cancer, joint injuries, even basic signs of aging. The only hitch: With one tiny exception, it isnt legal in the United States.

We all know the stem cell revolution is occurring outside the U.S., says Brian Mehling, M.D., a Manhattan-based orthopedic surgeon who is certainly doing his part to foment the insurgency. A coproducer of the film, as well as its charismatic recurring subject, Mehling is bringing stem cell tourism into the spotlight and determined to lift the curtain on a medical field that remains mysterious to most. His Blue Horizon medical clinics, with locations in China and Slovakiaand three more set to open in Mexico, Israel, and Jamaicacater to American tourists looking to cutting-edge therapy for help when traditional medicine fails.

Stem cells are the undifferentiated cells that abound in newborns and have the ability to transform into blood, nerve, or muscle cells and aid the body in self-repair. Proselytizers like Mehling say they constitute the latest in holistic medicine, allowing the body to healwithout drugs, surgery, or side effects. At clinics such as Mehlings, doctors either inject the cells, which are generally obtained from umbilical cords during C-sections, into a patients spinal cord (much like an epidural), or administer them via IV drip. The process is alarmingly quick, and patients can typically check out of the facility by the end of the day. One of the few stem-cell therapies approved for use in the United States is one used to treat the blood disease known as beta thalassemia; in that instance, the treatment replaces damaged blood in the immune system and saves tens of thousands of lives each year. Few other stem cell applications, however, have been proven effective in the rigorous clinical trials the Food and Drug Administration requires before signing off on any treatment.

In fact, stem cell clinics remain completely unregulated, and there have been incidents of related troubles. In one recent report , Jim Gass, a resident of San Diego who traveled to stem cell clinics in Mexico, China, and Argentina to help recover from a stroke, later discovered a sizable tumor on his spinal columnand the cancerous cells belonged to somebody else. Troubling cases also emerged at a loosely regulated clinic in Sunrise, Florida where, earlier this spring, three women suffering macular degeneration reported further loss of vision after having stem cells, extracted from their belly fat via liposuction, injected into their eyes. Though, on the whole, reports of treatments at clinics gone awry remain relatively few.

In his film, Stem Cells: The Next Frontier , which is set to appear at Cannes Film Festival this month, Mehling offers a persuasive side of the story, with rapturous testimonials from patients, some of whom who have regained the ability to walk after their stem cell vacations. Added bonus: They come home with better skin, bigger sex drive, and (in the case of at least one balding patient) more hair.

However compelling, there is scant evidence that the injections actually make a difference, and most American doctors caution against buying into the hype. Stem cell researcher Jaime Imitola, M.D. and Ph.D, director of the progressive multiple sclerosis clinic research program at Ohio State University, says he is impressed by the evidence that stem cells can help with neurological disorders in animals. But the question is how can you translate it into clinical trials? We still dont know what were doing when we put stem cells in people.

David Scadden, a professor of medicine and stem cell and regenerative biology at Harvard, and the director of Harvards Stem Cell Institute, says that stem cell tourism is a waste of money for the time being. A world-renowned expert in stem cell science, he remains optimistic about its future applications. Researchers are currently looking into reprogramming, for instance, which effectively converts a mature cell into a stem cell. You rewind its history so it forgets its a blood cell or a skin cell and it rewinds back in time and it can become any cell type, he says. Youd be able to test drugs on these cells, and it could be used to reverse Type 1 diabetes.

For now, though, he does not recommend experimenting with stem cells before we understand them well enough to properlyand safelyharness their benefits. People call me about it all the timethey say, I have this knee thats bugging me, Im going to one of these clinics, he says. His response? For the most part they dont do harm. But nobody Ive spoken with has come back to me and said, You Harvard docs have to get on this . . . . Not yet.

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Stem Cell Tourism Is the Controversial Subject of a New Cannes Documentary - Vogue.com

Blood study insight could improve stem cell therapy success – Medical Xpress

May 12, 2017

Researchers have pinpointed a key enzyme that is vital for the production of fresh blood cells in the body. The enzyme is essential for the survival of specialised stem cells that give rise to new blood cells, the study found. Experts say the findings could help to improve the success of stem cell therapies that are being developed to treat some blood cancers and disorders of the immune system.

Enzyme

Scientists focused on an enzyme called fumarase, which is known to play a key role in the generation of energy inside cells. Children with gene mutations that affect fumarase have blood defects, which prompted researchers to investigate its function.

The mouse study found that deletion of fumarase from blood cells causes major defects in new blood cell production. These defects could be traced back to defects in the specialised stem cells that give rise to new blood cells. Blocking the enzyme causes a molecule called fumarate to build up inside the cells, which has wide-ranging toxic effects.

Stem cells

The study sheds new light on the conditions that blood stem cells need to survive, which could help to boost the success of stem cell therapies, the researchers say. Stem cell metabolism is an emerging field of research with an immense therapeutic potential. In future, we hope to identify the biochemical pathways affected by fumarate in stem cells and, by manipulating these pathways, improve the success of stem cell transplant therapies.

Professor Kamil Kranc says, "The research was prompted when researchers noticed that children with genetic mutations in the fumarase gene have blood defects."

The research was prompted when researchers noticed that children with genetic mutations in the fumarase gene have blood defects.

Scientists from the Medical Research Council Centre for Regenerative Medicine at the University of Edinburgh led the study.

The research is published in the Journal of Experimental Medicine and was funded by The Kay Kendall Leukaemia Fund, Cancer Research UK, Bloodwise, Tenovus Scotland and Wellcome.

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Blood study insight could improve stem cell therapy success - Medical Xpress

Cromwell’s Biological Industries inks stem-cell training pact with Calif. Institute – Hartford Business

PHOTO | Steve Laschever

Biological Industries has a distribution-storage depot in Cromwell for its line of cell-culture media in which human and animal stem cells are grown.

Cromwell-based Biological Industries USA is partnering with a Monrovia, Calif.-based nonprofit stem cell institute to provide training for aspiring young scientists.

Biological Industries' partner, the Pathways to Stem Cell Science institute, will host early career scientists ranging from high school to college-level students and beyond and provide hands-on experience in culturing human pluripotent stem cells. Biological Industries will provide stem cell product and technology support through program scholarships for the Pathways to Stem Cell Science training programs.

The financial terms of the deal were not disclosed.

Biological Industries, which specializes in stem cell research, cellular reprogramming and regenerative medicine, is a division of Biological Industries Israel LTD. Pathways to Stem Cell Science programs provide classroom and laboratory-based courses in molecular cell biology, stem cell biology, and regenerative medicine to equip students with the skills to help them gain a competitive edge in the industry.

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Cromwell's Biological Industries inks stem-cell training pact with Calif. Institute - Hartford Business

Using single-cell RNA sequencing and clever statistical analysis to track stem cells as they mature – Phys.Org

May 11, 2017 The nose is lined with sensory tissue, the olfactory epithelium, that contains various types of cells, all of which arise from olfactory stem cells (green). Among these are smell-sensing neurons (orange), progenitor cells (cyan) and support cells (magenta sustentacular cells and blue microvillous cells). Credit: Russell Fletcher & John Ngai, UC Berkeley

Adult stem cells have the ability to transform into many types of cells, but tracing the path individual stem cells follow as they mature and identifying the molecules that trigger these fateful decisions are difficult in a living animal.

University of California, Berkeley, neuroscientists have now combined new techniques for sequencing the RNA in single cells with detailed statistical analysis to more easily track individual stem cells in the nose, uncovering clues that someday could help restore smell to those who have lost it.

The results are published this week in the journal Cell Stem Cell.

"A stem cell's job is twofold: to replace or recreate mature cells that are lost over time, both through normal aging and after injury, and to replace themselves so that the process can continue over the life of the animal," said senior author John Ngai, the Coates Family Professor of Neuroscience and a member of UC Berkeley's Helen Wills Neuroscience Institute and the Berkeley Stem Cell Center. "We are getting closer to understanding how mature sensory neurons are generated from olfactory stem cells, an understanding that's key for an eventual stem cell therapy to restore function."

Ngai noted that perhaps one-quarter of all people over the age of 50 have some loss of smell, yet doctors have little understanding why, and no treatments for most cases. There's not even a standardized test for loss of smell, as there is for vision or hearing loss, in spite of widespread reports of suffering by patients who have lost their sense of smell.

"Some cases of anosmiathe loss of the sense of smellare due to traumatic injury, and there is generally not a whole lot you can do about that," he said. "But some are age-related, or occur for reasons we don't quite know. In the case of age-related anosmias, it could be because the stem cells are just not doing their job replacing the cells that are naturally lost over time. One idea is that if we could harness the very stem cells that are in the noses of people who are losing smell, maybe we can figure out a way to restore function, by getting them to regenerate the cells that are lost."

Tracking cell fate

Ngai, who directs the Functional Genomics Laboratory in UC Berkeley's California Institute for Quantitative Biosciences, focuses on the cells and regulatory molecules involved in our sense of smell. Olfactory cells in the nose are unusual in that they are part of the body's outer layer, or epithelium, but also part of the nervous system, incorporating neurons that connect directly with the smell centers in the brain.

His group has been working with adult olfactory stem cells that give rise to the neurons that sense odors and other cells, such as sustentacular cells, that support the neurons. A new technique for sequencing the RNA in a single cell has been revolutionary, Ngai said, allowing researchers to trace which stem cells in a densely packed tissue become specialized, based on the mRNA present in the cell, which indicates which genes are being expressed. Nevertheless, it is difficult to follow stem cells that can potentially differentiate into different types of cells.

Ngai's group teamed up with UC Berkeley statisticians and computer scientists - led by Sandrine Dudoit, a professor of biotstatistics and statistics, Elizabeth Purdom, a professor of statistics, and Nir Yosef, a professor of electrical engineering and computer sciences - to develop a way to analyze the experimental data and identify cells with similar RNA profiles, indicative of specific cell types and developmental states.

As a result, the team was able to trace the paths that cells take as they turn into sustentacular cellswhich seems to be the default fate for olfactory stem cellsand into neurons and other types of cells. They also were able to identify a signaling pathway known as "Wnt" that triggers the olfactory stem cell to become a sensory neuron.

Ngai cautions that the immediate implications of the work are limited to animal models, which provide the necessary foundation for eventually addressing human anosmias. "But with this information, we now have a window into what controls the process and therefore a window into manipulating or coopting that process to stimulate regeneration" he said. "There has been a lot of work on Wnt signaling pathways, for example, so there are a lot of small-molecule drugs that could be tested to trigger a stem cell to mature into a neuron."

The sequencing and statistical techniques the team developed can also be used by others studying regulation of stem cells in other tissues, organ systems or organisms, he said.

Explore further: Neuroscientists find genetic trigger that makes stem cells differentiate in nose epithelia

University of California, Berkeley, neuroscientists have discovered a genetic trigger that makes the nose renew its smell sensors, providing hope for new therapies for people who have lost their sense of smell due to trauma ...

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Using single-cell RNA sequencing and clever statistical analysis to track stem cells as they mature - Phys.Org

Sniffing out stem cell fates in the nose – UC Berkeley

Adult stem cells have the ability to transform into many types of cells, but tracing the path individual stem cells follow as they mature and identifying the molecules that trigger these fateful decisions are difficult in a living animal.

Neurons (green) derived from a single stem cell in the olfactory epithelium of the nose. The magenta cells are support cells called sustentacular cells. (Russell Fletcher image)

UC Berkeley neuroscientists have now combined new techniques for sequencing the RNA in single cells with detailed statistical analysis to more easily track individual stem cells in the nose, uncovering clues that someday could help restore smell to those who have lost it.

The results are published this week in the journal Cell Stem Cell.

A stem cells job is twofold: to replace or recreate mature cells that are lost over time, both through normal aging and after injury, and to replace themselves so that the process can continue over the life of the animal, said senior author John Ngai, the Coates Family Professor of Neuroscience and a member of UC Berkeleys Helen Wills Neuroscience Institute and the Berkeley Stem Cell Center. We are getting closer to understanding how mature sensory neurons are generated from olfactory stem cells, an understanding thats key for an eventual stem cell therapy to restore function.

Ngai noted that perhaps one-quarter of all people over the age of 50 have some loss of smell, yet doctors have little understanding why, and no treatments for most cases. Theres not even a standardized test for loss of smell, as there is for vision or hearing loss, in spite of widespread reports of suffering by patients who have lost their sense of smell.

Some cases of anosmia the loss of the sense of smell are due to traumatic injury, and there is generally not a whole lot you can do about that, he said. But some are age-related, or occur for reasons we dont quite know. In the case of age-related anosmias, it could be because the stem cells are just not doing their job replacing the cells that are naturally lost over time. One idea is that if we could harness the very stem cells that are in the noses of people who are losing smell, maybe we can figure out a way to restore function, by getting them to regenerate the cells that are lost.

Tracking cell fate Ngai, who directs the Functional Genomics Laboratory in UC Berkeleys California Institute for Quantitative Biosciences, focuses on the cells and regulatory molecules involved in our sense of smell. Olfactory cells in the nose are unusual in that they are part of the bodys outer layer, or epithelium, but also part of the nervous system, incorporating neurons that connect directly with the smell centers in the brain.

The nose is lined with sensory tissue, the olfactory epithelium, that contains various types of cells, all of which arise from olfactory stem cells (green). Among these are smell-sensing neurons (orange), progenitor cells (cyan) and support cells (magenta sustentacular cells and blue microvillous cells). (Russell Fletcher image)

His group has been working with adult olfactory stem cells that give rise to the neurons that sense odors and other cells, such as sustentacular cells, that support the neurons. A new technique for sequencing the RNA in a single cell has been revolutionary, Ngai said, allowing researchers to trace which stem cells in a densely packed tissue become specialized, based on the mRNA present in the cell, which indicates which genes are being expressed. Nevertheless, it is difficult to follow stem cells that can potentially differentiate into different types of cells.

Ngais group teamed up with UC Berkeley statisticians and computer scientists led by Sandrine Dudoit, a professor of biotstatistics and statistics, Elizabeth Purdom, a professor of statistics, and Nir Yosef, a professor of electrical engineering and computer sciences to develop a way to analyze the experimental data and identify cells with similar RNA profiles, indicative of specific cell types and developmental states.

As a result, the team was able to trace the paths that cells take as they turn into sustentacular cells which seems to be the default fate for olfactory stem cells and into neurons and other types of cells. They also were able to identify a signaling pathway known as Wnt that triggers the olfactory stem cell to become a sensory neuron.

Ngai cautions that the immediate implications of the work are limited to animal models, which provide the necessary foundation for eventually addressing human anosmias. But with this information, we now have a window into what controls the process and therefore a window into manipulating or coopting that process to stimulate regeneration he said. There has been a lot of work on Wnt signaling pathways, for example, so there are a lot of small-molecule drugs that could be tested to trigger a stem cell to mature into a neuron.

The sequencing and statistical techniques the team developed can also be used by others studying regulation of stem cells in other tissues, organ systems or organisms, he said.

The work was spearheaded by senior postdoctoral researcher Russell Fletcher and graduate student Diya Das, and was funded by the National Institute on Deafness and Other Communications Disorders, the National Institute of Mental health, the National Institute on Aging, National Human Genome Research Institute, the National Center for Research Resources, the National Institute of General Medical Sciences, the California Institute of Regenerative Medicine and the Berkeley Siebel Stem Cell Center.

Other co-authors include Levi Gadye, Kelly Street, Ariane Baudhuin, Allon Wagner, Michael Cole, Quetzal Flores, Yoon Gi Choi and Davide Risso.

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Sniffing out stem cell fates in the nose - UC Berkeley

Stem cell therapy holds promise for treating most severe cases of … – Medical Xpress

May 11, 2017

An analysis of data from the entire development program consisting of three trials assessing the feasibility of using a stem cell therapy (CD34+ cells) to treat patients with the most severe cases of angina, refractory angina (RA), showed a statistically significant improvement in exercise time as well as a reduction in mortality. Results from "CD34+ Stem Cell Therapy Improves Exercise Time and Mortality in Refractory Angina: A Patient Level Meta-Analysis" were presented today as a late-breaking clinical trial at the Society for Cardiovascular Angiography and Interventions (SCAI) 2017 Scientific Sessions in New Orleans.

One of the warning signs of coronary artery disease is angina, or chest pain, which occurs when the heart muscle does not receive enough blood. Unlike angina pectoris or "stable angina," which can often be treated with medication, RA can be incapacitating, impacting quality of life. In the most severe cases, those with class III or IV angina, treatment options are exhausted, and patients remain severely debilitated. Unfortunately, one of the untoward consequences of the improved survival of patients with chronic ischemic heart disease is more patients with refractory angina.

A meta-analysis of three trials that each showed promising results looked at injecting RA patients with autologous CD34+ cellswhich have been shown to increase blood flowand the therapy's effect on mortality and total exercise time (TET), an important predictor of long-term mortality.

Data from 304 patients was extracted and analyzed from phase 1 (24 patients), ACT-34 and ACT-34 extension studies (168 patients), and RENEW (112 patients), which was prematurely terminated by the sponsor due to financial considerations.

"The goal of this meta-analysis was to combine patient level data from three very similar trials to try understand what it would tell us," said lead investigator Tom Povsic, MD, FSCAI, associate professor at the Duke Clinical Research Institute (DCRI) and an interventional cardiologist at Duke University School of Medicine.

Results showed that patients treated with CD34+ cell therapy (n=187) improved TET by 80.5 12.1, 101.8 13.7, and 90.5 14.7 seconds at three months, six months, and 12 months compared with 28.1 15.7, 48.8 18.2, and 39.5 20.3 seconds for the placebo group (n=89), resulting in treatment effects of 52.5 (p=0.002), 52.9 (p=0.009) and 50.9 (p=0.027) seconds.

The relative risk of angina was 0.90 (p=0.40), 0.81 (p=0.14), and 0.79 (p=0.17) at three months, six months, and 12 months in CD34+ treated patients.

CD34+ treatment decreased mortality by 24 months (2.6 percent vs. 11.8 percent, p=0.003). In addition, major adverse cardiac events were less frequent (29.8 percent for CD34+ patients vs. 40.0 percent for the placebo group, p=0.08).

"Therapies for these patients are direly needed," said Povsic, "and results from our meta-analysis are very compelling. Most importantly, the number of patients in our meta-analysis approximates those who were targetedfor enrollment in RENEW, the prematurely terminated phase III study. These results suggest that had RENEW been completed, a regenerative therapy for these patients might meet criteria for approval. I still think this therapy has a lot of promise."

Timothy Henry, MD, chief of cardiology at Cedars-Sinai Medical Center in Los Angeles, agrees "CD34+ cell therapy appears to be an extremely safe and effective therapy for this growing and challenging patient population with limited options."

Explore further: Stem cell therapy shows potential for difficult-to-treat RA patient population

More information: Povsic presented "CD34+ Stem Cell Therapy Improves Exercise Time and Mortality in Refractory Angina: A Patient Level Meta-Analysis" on Thursday, May 11, 2017 11:30 a.m. CDT

A study using a stem cell therapy to treat challenging refractory angina (RA) patients demonstrated promising results, including improved exercise time, reduced angina and reduced mortality. The RENEW results were presented ...

A two-year, multi-center clinical study with 167 patients with class III-IV refractory angina randomized to low and high dose CD34+ cells or placebo has revealed that patients who received either a high or low dose of CD34a ...

The absolute cumulative probability of death at 12 months was 5 percent lower for patients who received routine invasive coronary angiography and revascularization as indicated during an unstable angina admission compared ...

An injection of stem cells into the heart could offer hope to many of the 850,000 Americans whose chest pain doesn't subside even with medicine, angioplasty or surgery, according to a study in Circulation Research: Journal ...

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Stem cell therapy holds promise for treating most severe cases of ... - Medical Xpress

New Lung "Organoids" in a Dish Mimic Features of Full-Size Lung – Newswise (press release)


Newswise (press release)
New Lung "Organoids" in a Dish Mimic Features of Full-Size Lung
Newswise (press release)
Newswise New York, NY (May 11, 2017)New lung organoidstiny 3-D structures that mimic features of a full-sized lunghave been created from human pluripotent stem cells by researchers at Columbia University Medical Center (CUMC). The team ...

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New Lung "Organoids" in a Dish Mimic Features of Full-Size Lung - Newswise (press release)

Study Identifies Root Cause of Gray, Balding Hair – NBC 10 Philadelphia

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Scientists in North Texas have identified the cells that cause hair to turn gray and to go bald findings that could one day help identify possible treatments.

Researchers from the University of Texas Southewestern Medical Center accidentally stumbled upon the discovery while studying a rare genetic disease that causes tumors to grow on nerves.

"When we saw the mice that we were expecting to form a tumor turned gray, we were really excited!" said Dr. Lu Le, an associate professor of dermatology at UT Southwestern.

The researchers found that a protein called KROX20, more commonly associated with nerve development, switches on in skin cells that become the hair shaft.

These hair cells then produce another protein called stem cell factor (SCF).In mice, these two proteins turned out to be important for baldness and graying.

When researchers deleted theSCF gene in mice,the animals' hair turned white; when they deleted the cells that produce KROX20, the mice stopped growing hair and eventually went bald, according to the study.

"We were really excited because as a dermatologist, I treat patients with hair disease, so when we found the root cause of why hair turns gray and hair loss, we just cannot let it go," Le said.

More research is needed to understand if the process works similarly in humans, and Le and his colleagues plan to start studying it in people.

Le hopes that, armed with this knowledge, scientists can develop a topical compound or transplant the necessary gene to hair follicles to correct these cosmetic problems.

Researchers say the findings could one day also provide answers about why humans age in general as hair graying and hair loss are among the first signs of aging.

At Hair Revival Studio in Dallas, clients say the possibility of a treatment for gray hair and baldness sounds remarkable.

"Everyone wants to have a good head of hair. There's a lot of confidence that comes with that," said Brandon Stewart.

Published at 8:55 AM EDT on May 11, 2017 | Updated at 9:54 AM EDT on May 11, 2017

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Study Identifies Root Cause of Gray, Balding Hair - NBC 10 Philadelphia

Uncovering the genetic mechanisms driving embryonic development – Medical Xpress

May 11, 2017 by Kevin Mccullough Ali Shilatifard, PhD, the Robert Francis Furchgott Professor and chair of the Department of Biochemistry and Molecular Genetics, was the senior author of the study that explored the activation of Hox genes in early embryonic development.

A new Northwestern Medicine study, published in Genes and Development, has identified two DNA elements crucial to the activation of a set of genes that drive the early development of embryos, and which also play an important role in the development of cancer cells.

So-called Hox genes are a related group that control the body plan of a developing embryo; in humans, they regulate the orientation and structure of the vertebrae and spinal cord as well as the location and growth of limbs. Previously, however, the question of how Hox genes become activated, moving from a silent form to an active form, have been poorly-understood by scientists.

"Hox genes are not only crucial for the proper development of the embryo but also play essential roles in tumor formation and metastasis. Understanding the mechanisms that trigger the expression of Hox genes could help us develop novel therapeutic approaches against cancer," said first-author Kaixiang Cao, PhD, a postdoctoral fellow in the laboratory of Ali Shilatifard, PhD, the Robert Francis Furchgott Professor and chair of the Department of Biochemistry and Molecular Genetics.

In the study, the authors present several experiments that provide evidence for a model of embryonic development that utilizes multiple layers of regulation as a "fail-safe mechanism" to guarantee organisms develop properly.

First, the scientists identified two sequences of DNA, located in a so-called "gene desert" between functioning genes, and demonstrated how these sequences ensure activation of Hox genes.

Previously thought to be "junk DNA," the sequences of DNA found in gene deserts have recently been found to play important regulatory roles, and irregularities in these stretches of the genetic code have been associated with disease, including some forms of cancer.

After the scientists pinpointed these previously unidentified DNA sequences, named E1 and E2, they demonstrated they were acting as "shadow enhancers," and regulated the early expression of Hox genes.

Utilizing mouse embryonic stem cell models that had been modified to lack one or both of the sequences, the scientists showed that the two sequences worked redundantly: deletion of either the E1 or E2 sequence resulted in unaffected activation, but removing both E1 and E2 stopped the Hox genes from activating properly.

Separately, the scientists also demonstrated that a protein called SET1A, part of a family of enzymes called COMPASS, which have previously been shown to activate Hox genes, also regulates Hox gene activation: without SET1A, several Hox genes failed to activate.

According to the scientists, the E1/E2 regulation and the SET1A regulation of Hox genes appear to be independent of each other, and are part of a series of multiple regulatory processes that work together to fine-tune the activation of genes essential for the early growth of embryos.

"Future studies that identify small molecules targeting SET1A and factors functioning through the E1/E2 DNA sequences will be important for developing therapies for Hox gene disorders," Shilatifard said.

The project's insight into the process by which Hox genes are regulated, has the potential to identify targets for new treatments for developmental diseases caused by dysfunction in Hox genes, as well as forms of cancer that arise from Hox gene errors, according to the authors.

Explore further: Molecular 'on switch' could point to treatments for pediatric brain tumor

More information: Kaixiang Cao et al. SET1A/COMPASS and shadow enhancers in the regulation of homeotic gene expression, Genes & Development (2017). DOI: 10.1101/gad.294744.116

A landmark study led by UNC School of Medicine researchers has identified the first genetic locus for anorexia nervosa and has revealed that there may also be metabolic underpinnings to this potentially deadly illness.

When a group of researchers in the Undiagnosed Disease Network at Baylor College of Medicine realized they were spending days combing through databases searching for information regarding gene variants, they decided to do ...

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Reported in Nature today, one of the largest sets of high quality human induced pluripotent stem cell lines from healthy individuals has been produced by a consortium involving the Wellcome Trust Sanger Institute. Comprehensively ...

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Over the last decade, it has made good sense to study the genetic drivers of cancer by sequencing a tiny portion of the human genome called the exome - the 2% of our three billion base pairs that "spell out" the 21,000 genes ...

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Uncovering the genetic mechanisms driving embryonic development - Medical Xpress