Category Archives: Gene Medicine

When Nisa Leung was pregnant with her first child in 2012, her doctor in Hong Kong offered her a choice. She could take a prenatal test that would require inserting a needle into her uterus, or pay $130 more for an exam that would draw a little blood from her arm.

Leung opted for the simpler and less risky test, which analyzed bits of the baby's DNA that had made its way into her bloodstream. Then Leung went on to do what she often does when she recognizes a good product: look around for companies to invest in.

The managing partner at Qiming Venture Partners decided to put money into Chinese genetic testing firm Berry Genomics, which eventually entered into a partnership with the Hong Kong-based inventor of the blood test. Over the next few months, Berry is expected to be absorbed into a Chinese developer in a $625 million reverse merger. And Leung's venture capital firm would be the latest to benefit from a boom in so-called precision medicine, an emerging field that includes everything from genetic prenatal tests to customizing treatments for cancer patients.

China has made the precision medicine field a focus of its 13th five-year plan, and its companies have been embarking on ambitious efforts to collect a vast trove of genetic and health data, researching how to identify cancer markers in blood, and launching consumer technologies that aim to tap potentially life-saving information. The push offers insight into China's growing ambitions in science and biotechnology, areas where it has traditionally lagged developed nations like the U.S.

"Investing in precision medicine is definitely the trend," said Leung, who's led investments in more than 60 Chinese health-care companies in the past decade. "As China eyes becoming a biotechnology powerhouse globally, this is an area we will venture into for sure and hopefully be at the forefront globally."

New Chinese firms like iCarbonX and WuXi NextCode that offer consumers ways to learn more about their bodies through clues from their genetic make up are gaining popularity. Chinese entrepreneurs and scientists are also aiming to dominate the market for complex new procedures like liquid biopsy tests, which would allow for cancer testing through key indicators in the blood.

Such research efforts are still in early stages worldwide. But doctors see a future beyond basic commercial applications, aiming instead for drugs and treatment plans tailored to a person's unique genetic code and environmental exposure, such as diet and infections.

Isaac Kohane, a bioinformatics professor at Harvard University, says when it comes to precision medicine, the science community has "Google maps envy." Just as the search engine has transformed the notion of geography by adding restaurants, weather and other locators, more details on patients can give doctors a better picture on how to treat diseases.

For cancer patients, for example, precision medicine might allow oncologists to spot specific mutations in a tumor. For many people with rare ailments like muscle diseases or those that cause seizures, it allows for earlier diagnosis. Pregnant women, using the kind of tests that Leung used, could also learn more about the potential for a child to inherit a genetic disease.

The global interest in the field comes as the cost of sequencing DNA, or analyzing genetic information, is falling sharply. But a number of hurdles remain. Relying on just genes isn't enough, and there must also be background information on a patient's lifestyle and medication history.

Precision medicine applications also require heavy investment to store large amounts of information. A whole genome is more than 100 gigabytes, according to an e-mailed response to questions from Edward Farmer, WuXi NextCode's vice president of communications and new ventures. "So you can imagine that analyzing thousands or hundreds of thousands of genomes is a true big data challenge."

WuXi NextCode was formed after Shanghai-based contract research giant WuXi AppTec Inc. acquired genomic analysis firm NextCode Health, a spin-off from Reykjavik, Iceland-based Decode Genetics, which has databases on the island's population. Wuxi NextCode continues to have an office in Iceland, where the population is relatively homogenous and therefore good for gene discovery.

"Genomics today is like the computer industry in the '70s," said Hannes Smarason, WuXi NextCode's co-founder and chief operating officer. "We've made great progress but there's still a long way to go."

In China, Wuxi NextCode now offers consumers genetic tests that cost between about $360 and $1,160, providing more details on rare conditions a child might be suffering from or even the risk of passing on an inherited disease.

China is diverse, and with 1.4 billion people, the planet's most populous nation. WuXi NextCode announced a partnership with Huawei Technologies Co., China's largest telecommunications equipment maker, in May to enable different institutions and researchers to store their data.

The goal is to use that deep pool of information -- which ranges from genome sequences to treatment regimens -- to find more clues on tackling diseases. WuXi says that "this will in many instances enable the largest studies ever undertaken in many diseases."

Another Chinese player, iCarbonX, which received a $200 million investment from Tencent Holdings Ltd. and other investors in April, is valued at more than $1 billion. It announced last month that it had invested $400 million in several health data companies to enable the use of algorithms to analyze reams of genomic, physiological and behavioral data to provide customized medical advice directly to consumers through an app.

The global precision medicine market was estimated to be worth $56 billion in revenue at the end of 2016, with China holding about 4 percent to 8 percent of the global market, according to a December report from Persistence Market Research.

Encouraging interventions for some patients too early, even before they have life-threatening diseases, comes with risks and ethical questions, Laura Nelson Carney, an analyst at Sanford C Bernstein, wrote in a Jan. 6 note. Still, precision medicine research has many benefits, and some in China see the country's push as a significant opportunity "to scientifically leapfrog the West," she said.

In the U.S., universities, the National Institutes of Health and American drugmakers are part of a broad march into precision medicine.

Amgen Inc. bought Icelandic biotechnology company DeCode Genetics for $415 million in 2012, to acquire its massive database on Iceland's population. U.S.-based Genentech Inc. is collaborating with Silicon Valley startup 23andMe to study the genetic underpinnings of Parkinson's disease.

"Humans are computable," said Wang Jun, the chief executive officer of China's iCarbonX. "So we need a computable model that we can use to intervene and change people's status, that's the whole point."

SundayMonday Business on 02/13/2017

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February 13, 2017 A 3-D map ofliver and pancreatic buds in a mouse embryo. Cells of the pancreas are marked in red and green, while liver cells appear in blue. Credit: Francesca Spagnoli, MDC

It is now possible to reprogram cells from the liver into the precursor cells that give rise to the pancreas by altering the activity of a single gene. A team of researchers at the Max Delbrck Center for Molecular Medicine in the Helmholtz Association (MDC) has now accomplished this feat in mice. Their results should make it feasible to help diabetic patients through cell therapy.

In patients suffering from type I diabetes, their immune system turns against their own bodies and destroys a type of pancreatic cell called islet cells. Without these cells, the pancreas is unable to produce the hormone insulin and blood glucose rises, which leads to diabetic disease. At that point, such patients need to inject insulin for the rest of their lives.

A way to provide a lasting help to the afflicted may be to grow new pancreatic cells outside of the body. MDC group leader and researcher Dr. Francesca has been pursuing the idea of reprogramming liver cells to become pancreatic cells. Dr. Spagnoli's team has now succeeded in thrusting liver cells into an "identity crisis"in other words, to reprogram them to take on a less specialized stateand then stimulate their development into cells with pancreatic properties.

Promising success in animal experiments

A gene called TGIF2 plays a crucial role in the process. TGIF2 is active in the tissue of the pancreas but not in the liver. For the current study Dr. Nuria Cerda Esteban, at the time a PhD student in Dr. Spagnoli's lab, tested how cells from mouse liver behave when they are given additional copies of the TGIF2 gene.

In the experiment, cells first lost their hepatic (liver) properties, then acquired properties of the pancreas. The researchers transplanted the modified cells into diabetic mice. Soon after this intervention, the animals' blood glucose levels improved, indicating that the cells indeed were replacing the functions of the lost islet cells. The results bring cell therapies for human diabetic patients one step closer to reality.

The obvious next step is to translate the findings from the mouse to humans. The Spagnoli lab is currently testing the strategy on human liver cells in a project funded in 2015 by the European Research Council. "There are differences between mice and humans, which we still have to overcome," Spagnoli says. "But we are well on the path to developing a 'proof of concept' for future therapies."

Explore further: Normal insulin rhythm restored in mouse pancreas cells by glucose pulse

More information: Nuria Cerd-Esteban et al. (2017): "Stepwise reprogramming of liver cells to a pancreas progenitor state by the transcriptional regulator Tgif2." Nature Communications. DOI: 10.1038/ncomms14127

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The significant role of beta cell 'hubs' in the pancreas has been demonstrated for the first time, suggesting that diabetes may due to the failure of a privileged few cells, rather than the behaviour of all cells.

It is now possible to reprogram cells from the liver into the precursor cells that give rise to the pancreas by altering the activity of a single gene. A team of researchers at the Max Delbrck Center for Molecular Medicine ...

A new study by researchers at King's College London has found that patients with diabetes suffering from the early stages of kidney disease have a deficiency of the protective 'anti-ageing' hormone, Klotho.

Latino children who live in areas with higher levels of air pollution have a heightened risk of developing Type 2 diabetes, according to a new USC-led study.

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Inducing an identity crisis in liver cells may help diabetics - Medical Xpress

VATICAN CITY The Vatican has issued an updated version of their charter for healthcare workers, removing question marks from modern ethical concerns such as euthanasia and the creation of human-animal chimeras by offering a clear set of guidelines.

In the past 20 years there have been two situations, two events that have made the production of a new healthcare charter necessary, Professor Antonio Gioacchino Spagnolo told CNA Feb. 6.

The first, he said, is scientific progress. In these 20 years there has been a lot of scientific progress in the field of the beginning of life as well as in the phase of the end of life, in the context of living.

But alongside advancements in science the Churchs Magisterium has also produced several texts dealing with new and current issues, offering an authoritative take on how they should be handled.

The charter, he said, encompasses a sort of collection of the various positions there have been, the various pronouncements, keeping the progress of bio-medicine in mind.

Spagnolo, director of the Institute of Bioethics and Medical Humanities at the Faculty of Medicine and Surgery at the Catholic University of the Sacred Heart in Rome, spoke to journalists at the Feb. 6 presentation of the new charter, and played a key role in drafting the new text.

A first edition of the charter was published in 1994, but in the wake of broad scientific advancements and various updates in the Churchs Magisterium, the Holy See Monday rolled out the new version of the charter for healthcare workers.

Released to coincide with the annual World Day of the Sick celebrations taking place in Lourdes, the updated charter includes all magisterial documents published since 1994 and will be sent to bishops conferences around the world.

At roughly 150 pages including the index, the charter is structured much like the old edition, and is divided into three parts: Procreation, Life, and Death.

The section on procreation covers everything from contraception, IVF, and the scientific use of embryos, including freezing them, as well as newer topics such as the mixing of human and animal gametes, the gestation of human embryos in animal or artificial wombs, cloning, asexual reproduction, and parthenogenesis.

In the Life section, topics covered are all of the health events that are in some way connected to living, Spagnolo said, including vaccinations, preventative care, drug testing, transplants, abortion, anencephalic fetuses, as well as gene therapy and regenerative medicine.

The social part of the charter also covers areas specifically linked to poverty, such as access to medicines and the availability of new technologies in developing countries or countries that are politically and economically unstable. Rare and neglected diseases are also covered in the new text.

In his comments to CNA, Spagnolo commented on recent cases the new, updated charter would cover, including the creation of human-pig chimeras, as well as the case of an elderly woman with dementia who was held down by her family during a euthanasia procedure.

The first case refers to the recent high-level scientific research project that culminated in the creation of chimeras, or organisms made from two different species.

While the project initially began by conducting the experiment on rats and mice, at the end of January it culminated with the human-pig mix, marking the first time a case had been reported in which human stem cells had begun to grow inside another species.

In the experiment, which appeared in the scientific journal Cell, researchers from various institutes, including Stanford and the Salk Institute in California, injected pig embryos with human stem cells when there were just a few days old and monitored their development for 28 days to see if more human cells would be generated.

Human cells inside a number of the embryos had begun to develop into specialized tissue precursors, however, the success rate of the human cells overall was low, with the majority failing to produce human cells.

Commenting on the case, Spagnolo said this type of hybridization between human and animal cells was primarily done to garner more scientific information. Its important that this research is done, he said, but cautioned that we cant be indifferent to how the information is used.

If a scientist decides to mingle human cells with those of another species in order to create some sort of hybrid being, this is of course something that cant be accepted because in some way it means using the generation of a life as an instrument to reach ones own ends.

However, if its done for a purpose other than generating alternate beings, such as growing human organs for transplant, Spagnolo said this would be acceptable.

One thing thats already being proposed, he said, is the possibility of xenografts, i.e. tissue grafts or organ transplants from a donor that is a different species than the recipient.

The idea of doing this, Spagnolo said, is to inoculate pigs with human cells, allowing the organs of the pig to receive human antigens, so when a transplant is done with a liver or heart from the pig inside a (human being), there wouldnt be the rejection that there is normally doing it with other species.

Spagnolo said that using the hybrid cells for organ or tissue transplant is acceptable because to transfer a human cell to a pig doesnt mean creating a life.

Rather, it allows the pig to have a genetic patrimony similar to that of a human being to then be able to use the organs to help people, he said, emphasizing the fact that its not pig cells being injected into human beings, but vice versa.

So to make a good, informed decision involves first of all seeing what type of experiments are being done, deciding from that whether its acceptable or not, then looking at what one intends to produce, what are the objectives one intends to reach.

Pointing to another touchy scientific case that came up recently when an elderly woman in her 80s was held down by her relatives as her doctors euthanized her, Spagnolo said this is the type of murky water which advanced statements or living wills wade into in countries where euthanasia and assisted suicide are legal.

The woman, who lived in the Netherlands, had dementia and had reportedly expressed a desire for euthanasia when the time was right at an earlier date, but had not done so recently.

When the woman began exhibiting fear and anger and was sometimes found to be wandering the halls of her nursing home, the senior doctor at the home determined that the womans condition meant the time was right, and put a sleep-inducing drug into her coffee so he could administer the lethal injection.

The woman was not consulted, and woke up as the doctor was trying to give the injection. When she fought the procedure, her family members were asked to hold her down while the injection was completed.

When medicine no longer does what it should because in a living will someone expresses their desire for assisted suicide, this statement completely alters the doctor-patient relationship, Spagnolo said.

He pointed to a bill that is currently on the table in Italy that would effectively legalize euthanasia and assisted suicide, requiring doctors to act on the advanced statements of their patients in this regard, and prohibiting them from conscientious objection.

This bill, as well as the case of the woman in the Netherlands, illustrates the difficulty of advance statements, Spagnolo said, explaining that if someone makes an advance statement and later decides against it, the fact of having said it before is used and is done (by) drugging the patient.

While the doctor-patient relationship is always a key element of the discussion, Spagnolo noted that various studies have been conducted showing a doctors behavior toward patients differs based on whether or not the patient has an advanced statement, specifically on euthanasia.

This disparity should be avoided. The doctor should always act the same way when the person is concerned, he said.

So with the new charter, all healthcare workers will now have a point of reference for some of these sticky scenarios, he said.

They can know that some things must be done, they are obligatory. Others, however, are only possibilities.

In this sense, the will of the patient is very important, not in the perspective of anticipating death, but in the perspective of knowing whether or not to accept and support certain interventions the doctor can do, but which the patient might think unsuitable.

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Vatican unveils updated healthcare charter as new ethical questions arise - Crux: Covering all things Catholic

1102 Bates Street, Suite 1670

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The GMP Facilities at the Center for Cell and Gene Therapy at Baylor College of Medicine are among the largest and most modern in the United States. Manufacturing of therapeutic biologics has existed at Baylor for more than twenty years, and is now centralized in 19,000 square feet on the 16th floor of the Feigin Center at Texas Childrens Hospital.

This facility opened in 2010 and contains 22 ISO 7 clean room suites supported by dedicated space for cell and product cold storage, flow cytometric analysis, quality control testing, data management and storage and quality assurance activities. The staff has extensive experience in GMP manufacturing of a wide variety of products and intermediates for cellular therapies and of viral and non-viral vectors, and master and working cell banks.

Manufacturing and testing supports more than 30 investigator-sponsored INDs conducted at the Texas Medical Center and institutions around the United States. Products have also been prepared under contract for researchers in Europe, Asia and Australia. The range of products that have been manufactured is shown under the Vector and Cell Therapy Facilitypages.

The facility is also accredited by the Foundation for the Accreditation of Cellular Therapy (FACT)and is CLIA registered for high complexity testing.

Center for Cell & Gene Therapy - cGMP Facilities

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Medical schools steadily improve clinical care with research Crain's Detroit Business ... test novel drugs and medical devices that range from UM's study on whether intravenous delivery of nutrients into the first part of the intestine or stomach will reduce eating and improve weight-related conditions to Wayne State's novel gene ...

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

U.S. Army scientists, working with medical technology companies, have successfully tested and used products and techniques that have enabled Army surgeons to replace the severely burned skin of soldiers as well as transplant new hands and even faces.

At Duke University, researchers are studying zebra fish to learn how science and medicine might someday be able to regenerate severed human spinal cords.

These examples one already in practice and the other in the early research stages illustrate the potential that regenerative medicine offers for the future of medical care.

This research aims to go beyond easing the pain of life-threatening illnesses by changing the way diseases affect the body and then eradicating them.

The vast majority of currently available treatments for chronic and/or life-threatening diseases are palliative, Morrie Ruffin, managing director of the Alliance for Regenerative Medicine (ARM), told Healthline.

ARM, based in Washington, D.C., is considered the preeminent global advocate for regenerative and advanced therapies.

Other treatments delay disease progression and the onset of complications associated with the underlying illness, he said. Very few therapies in use today are capable of curing or significantly changing the course of disease.

Regenerative medicine has the unique ability to alter the fundamental mechanisms of disease, and thereby offer treatment options to patients where there is significant unmet medical need.

And it has the potential to address the underlying causes of disease, Ruffin said, representing a new and growing paradigm in human health.

The field encompasses a number of different technologies, including cell, gene, and tissue-based therapies.

Read more: Re-growing teeth and healing wounds without scars

With the Army breakthroughs, government investment was key.

The U.S. Department of Defense (DOD) has invested more than $250 million in regenerative medicine research over the past decade in an effort to make promising technologies available to wounded service members.

Dr. Wendy Dean is medical officer for the Tissue Injury and Regenerative Medicine Project Management Office at the U.S. Army Medical Materiel Development Activity at Fort Detrick, Md., home to the Armys Medical Research and Materiel Command.

Those investments have yielded a stress-shielding surgical bandage, Embrace, to reduce scarring after surgery, Dean told Healthline. The research has also enabled tremendous progress in burn care, allowing surgeons to improve recovery from severe burns with the use of novel skin replacement strategies, such as ReCell spray-on skin, or skin substitutes such as StrataGraft. These skin replacement methods reduce or eliminate the need for donor sites, a frequent request of burn patients.

These revolutionary products were not developed by the Army, Dean said, but were supported with research funding, initially through the Armed Forces Institute of Regenerative Medicine.

The DOD also has invested in hand and face transplantation efforts for service members and civilians whose injuries are so severe that conventional reconstruction is insufficient, she said.

Dean noted that DOD funding has supported 13 hand transplants to date, including a transplant for retired Sgt. Brendan Marrocco in 2012. He was the first service member to survive quadrilateral amputations sustained in combat. The funding also supported eight face transplants.

The Armys goal is to heal those injured in battle.

Regenerative medicine is still young, but it has shown tremendous progress over the last decade, Dean said. Our mission is to make wounded warriors whole by restoring form, function, and appearance. This field offers the best hope to someday fully restore lost tissue with tissue that is structurally, functionally, and aesthetically a perfect match. It may be years before the vision is a widespread reality, but the field is well on its way.

Read more: Regenerative medicine doctor says forget the pills

At Duke University, Kenneth Poss, professor of cell biology, and director of the Regeneration Next initiative, was the senior investigator for a study of spinal cord regeneration in zebra fish.

Those findings were published in November in the journal ScienceDaily.

In my lab, we are researching genetic factors that enable regeneration of tissues such as heart and spinal in nonmammalian animals like zebra fish, Poss told Healthline. A scientist in my lab, Mayssa Mokalled, led a study finding that a gene called connective tissue growth factor [CTGF] is important for spinal cord regeneration in zebra fish after an injury that completely severs the cord.

CTGF is necessary to stimulate cells called glia to form a tissue bridge across the severed parts of the spinal cord an early step in spinal cord regeneration.

Within eight weeks, the scientists found that zebra fish regenerate a severed spinal cord, including nerve cells, and fully reverse their paralysis.

Developing techniques to treat and reverse spinal cord damage, a paralyzing and often fatal injury, is a pressing need in regenerative medicine, Poss said.

Our findings present a step toward understanding which glial cells can be encouraged to help heal the spinal cord, and how to stimulate this activity, he said. This is just the first step in many before the findings could be applied to humans.

Poss is already planning trials with mice that he hopes to start in the next few months. Mice represent an important stage in applying his latest findings, he said.

Read more: Should you store or donate your childs umbilical cord blood?

So, why is regenerative medicine important?

Regenerative medicine seeks ways to re-grow or engineer healthy tissue without the need for transplants, Poss said. On a global scale, theres a tremendous organ shortage, and transplantation is an expensive and nonpermanent solution.

Imagine the number of lives that could be improved if, for example, we could find ways to use the bodys innate healing mechanisms to regenerate heart muscle in patients that are spiraling toward heart failure after a heart attack.

Imagine how many lives could be improved if we could find interventions that restore functional spinal cord tissue and reverse paralysis.

Ruffin of ARM sees a promising future for regenerative medicine.

We will continue to see the development of additional regenerative medicine therapies for a broad number of acute and chronic, inherited and acquired diseases and disorders, he said. Therapies in this area will continue to advance along the regulatory pathway, many of which are entering phase III clinical trials this year.

In fact, in the next two years, we are anticipating a number of U.S. and E.U. approvals in the cell and gene therapy sector, including therapies that address certain types of cancers, debilitating retinal disorders, rare genetic diseases, and autoimmune conditions. We also expect to see sustained investment, which will help fuel growth and product development within this sector.

A number of cell and gene therapies and technology platforms are demonstrating real potential to address areas of significant unmet medical need, Ruffin said.

These include cell therapies for blood cancers and solid tumors; gene therapies for rare genetic diseases as well as chronic conditions; and gene editing for the precise targeting and modification of genetic material of a patients cells to cure a broad range of diseases with a single treatment.

Poss at Duke talked about the ultimate quest.

Regenerative medicine has been most successful in restoring or replacing the hematopoietic tissue that creates blood, he said.

We still lack successful regenerative therapies for most tissues, Poss said. The future of regenerative medicine the holy grail will be stimulating the regeneration of healthy tissue in patients without adding cells or manufactured tissue.

Working out the details of innate mechanisms of regeneration in animals like salamanders, zebra fish, and mice, can inform this approach, he said. So can improvement in factor delivery and genome editing applications to encourage the regeneration of healthy tissue.

Ultimately, Poss said, regenerative medicine will change the toolbox of physicians and surgeons, with major impact on outcomes of diabetes, spinal cord injuries, neurodegenerative disease, and heart failure.

ARM says the public does not realize how far the field has progressed in recent years.

Currently, there are more than 20 regenerative medicine products on the market, Ruffin said, primarily in the therapeutic areas of oncology, musculoskeletal and cardiovascular repair, and wound healing.

More than 800 clinical trials are now underway to evaluate regenerative advanced therapies in a vast array of therapeutic categories, he said.

Were seeing a significant focus on oncology, cardiovascular disease, and neurodegenerative diseases, with more than 60 percent of trials falling into one of these three categories, he added. Even though the majority of people perceive regenerative medicine as something of the future, its actually here and now.

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Regenerative Medicine Has a Bright Future - Healthline

New method of genetic engineering indispensable tool in ... Science Daily Scientists are pioneering a new method of genetic engineering for basic and applied biological research and medicine. Their work has the potential to open new ... and more »

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New method of genetic engineering indispensable tool in ... - Science Daily

Genetic profiling can guide stem cell transplantation for patients with myelodysplastic syndrome, study finds Science Daily A single blood test and basic information about a patient's medical status can indicate which patients with myelodysplastic syndrome (MDS) are likely to benefit from a stem cell transplant, and the intensity of pre-transplant chemotherapy and/or ... and more »

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February 10, 2017

An article published in Experimental Biology and Medicine (Volume 242, Issue 3, February, 2017) identifies microRNAs (miRNAs) as key factors in some hemoglobinopathies, genetic disorders characterized by alterations in the level or structure of the globin proteins that are responsible for oxygen transport in the blood. The study, led by Dr. Thais Fornari, from the Department of Internal Medicine at the University of Campinas in Brazil demonstrated that differential expression of miRNAs may be responsible for the variations in globin gene expression observed in patients with two hemoglobinopathies: hereditary persistence of fetal hemoglobin deletion type 2 (HPFH-2) and Sicilian-thalassemia.

HPFH-2 and Sicilian-thalassemia are conditions described as large deletions of the human -like globin cluster, with no -globin expression and compensatory increases in -globin expression. MicroRNAs (miRNAs) are small non-coding RNAs that participate in a wide range of biological processes including erythropoiesis. miRNAs silence the expression of other genes by binding to their mRNAs, and blocking protein synthesis and/or initiating mRNA degradation. Transcription factors such as BCL11A and SOX6, which regulate -globin gene expression, are potential targets for several microRNAs based on in silico analysis. Thus, novel miRNA-mediated pathways may explain the differences in the expressions of -globin in Sicilian thalassemia and HPFH-2.

In the current study, Dr. Fornari and colleagues compared the miRNA profiles of erythroid cells derived from individuals heterozygous for HPFH-2 and Sicilian-thalassemia. Forty-nine differentially expressed miRNAs that may participate in -globin gene regulation and red blood cell function were identified. Twelve of these miRNAs potentially targeted the BCL11A gene, and down-regulation of BCL11A gene expression in HPFH-2 was verified by qPCR. This research suggests an important action of miRNAs in the regulation of globin expression in patients. Fornari said that these findings "may partially explain the phenotypic differences between HPFH-2 and Sicilian -thalassemia and the variable increases in -globin gene expression in these conditions. Moreover, these data support erythroid BCL11A as a therapeutic target for sickle cell disease and -thalassemia major patients."

Dr. Steven R. Goodman, editor-in-chief of Experimental Biology and Medicine, said, "Fornari and colleagues provide further evidence for the role of miRNA networks in the regulation of fetal hemoglobin expression, via altered expression of BCL11A and SOX6. These studies are important when considering these transcription factors as potential therapeutic targets".

Explore further: Mechanisms, therapeutic targets of microRNA-associated chemoresistance in epithelial ovarian cancer

Journal reference: Experimental Biology and Medicine

Provided by: Experimental Biology and Medicine

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Researchers find potential treatments for hemoglobinopathies - Medical Xpress

Using stem cells and gene therapy to treat orcure disease may still sound like science fiction, but a scientific meeting here last week emphasizedall the fronts onwhich it is moving closer and closer to fact.

Were entering a new era in medicine, said Lloyd Minor, MD, dean of the School of Medicine, in his opening remarks at the first annual symposium of the schools new Center for Definitive and Curative Medicine. Stanford researchersare poised to use stem cells and gene therapy to amelioratea wide swath of diseases, from common diagnoses such as diabetes and cancerto rare diseases ofthe brain, blood, skin, immune system and other organs. Ultimately, the goal is to create one-time treatments that can provide lifetime cures; hence the definitive and curative part of the centers name. Stanford is a leader in this branch of medical research, Minor said, addingThis is a vital component of our vision for precision health.

Stanford has a long history of leading basic-science discoveries in stem cell biology, andis now engaged in studyingmany different ways those discoveries couldbenefit patients, saidMaria Grazia Roncarolo, MD, who leads the new center.Our job is to produce clinical data so compelling that industry will pick up the product and take it to the next stage, Roncaraolo told the audience.

Among otherevent highlights:

More coverage of the days events is available in a story from the San Jose Mercury News that describeshowAnthonyOro, MD, PhD, and his colleagues are fighting epidermolysis bullosa, a devastating genetic disease of the skin. Oro closed his talk with a slightly goofy photo of a man getting a spray tan. It got a laugh, but his point was serious: Our goal for the cell therapy of the future is spray-on skin to correct a horrible genetic disease.

Ambitious? Yes. Science fiction? In the future, maybe not.

Previously: One of the most promising minds of his generation: Joseph Wu takes stem cells to heart,Life with epidermolysis bullosa: Pain is my reality, pain is my normaland Rat-grown mouse pancreases reverse diabetes in mice, say researchers Photo of Matthew Porteus courtesy of Stanford Childrens

Read more from the original source:
Stanford scientists describe stem-cell and gene-therapy advances in scientific symposium - Scope (blog)