Archive for February, 2015

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Newswise A first of its kind genetic study confirms the history of the Druze community: The community began to form genetically in the 11th century AD, and there has since been no genetic impact of other ethnic groups on the community. This is according to a new study conducted by a team of researchers led by Prof. Gil Atzmon of the University of Haifa, Prof. Jamal Zidan of the Ziv Medical Center, Zefat, and Prof. Eitan Friedman of the Chaim Sheba Medical Center, Tel Hashomer. This is the first genetic study to discover that the Druze community has genetic origins in the 11th century AD, said Professor Atzmon of the University of Haifa. This genetic finding correlates with the Druze communitys beliefs regarding their origin.

Traditionally, the Druze people believe that their community was founded in the 11th century AD as a new religious movement under the sixth caliph of the Fatimid Dynasty of Egypt. There are currently 1.5 million Druze around the world, residing mainly in Syria and Lebanon, with the remainder in Israel and Jordan. According to Druze tradition, marriages take place only within the Druze community.

An international team of researchers was formed to perform this current study, published in the European Journal of Human Genetics Nature, which sought to examine whether the Druze people of today have a similar gene pool and if so, when that gene pool began to take shape. The head of the team, Prof. Atzmon of the University of Haifas Department of Human Biology and of the Department of Medicine and Genetics, the Albert Einstein College of Medicine, NY, together with Prof. Zidan, the director of the oncology department at Ziv Medical Center and of the Faculty of Medicine in the Galilee, Bar-Ilan University and Prof. Eitan Friedman of the Sackler School of Medicine, Tel Aviv University, were joined by Dr. Dan Ben-Avraham of the Department of Medicine and Genetics, Albert Einstein College of Medicine, NY, Dr. Shai Carmi of the Department of Computer Science, Columbia University, NY, and Dr. Taiseer Maray of the organization, Golan for Development.

The study included 120 participants from forty families. Twenty families were from the village of Beit Jan located in the Upper Galilee and twenty were from Majdal Shams, in the Golan Heights. The families were selected according to the origins of their extended families (clans), based on their family names and on information that was passed down orally from generation to generation. The mother, father and son of each family were genetically tested. All the families who participated in the study were from different clans so that the sample would be representative and it excluded first- or second-degree family relationships to any other participants in the study. These characteristics all significantly increased the studys genetic accuracy. In this study, we incorporated data that was published on the Druze of Lebanon, the Carmel Mountain region and various other populations in order to test the genetic structure of the Druze population relative to other populations, said Prof. Zidan.

The results indicated that the Druze do indeed share a high genetic similarity that significantly distinguishes them from member of other groups and communities in the Middle East. When the researchers went back in time to discover when this genetic similarity began, they reached the 11th century AD, about 22-47 generations ago (there are differences of opinion over the duration of a generation). During this period a genetic bottleneck was formed, i.e., the genetic origin of many descendants came to an end, the communitys population decreased and the individuals in the population became more alike genetically. According to Prof. Atzmon, their research findings limit the ancestors of the Druze community to several hundred families, who founded the community in the 11th century AD. The researchers also found that there is no evidence of new genes entering the Druze gene pool over the last 1,000 years. In other words, no additional groups from the outside joined this community. In addition, the researchers found evidence of genetic differences between Druze populations from different regions: Lebanon, the Golan Hights, the Upper Galilee and the Carmel Mountain. This strengthens the evidence that marriages take place only within each clan.

When they went further back in time, the researchers discovered another interesting finding. It came to light that, 500 years prior to the beginning of the Druze religion, around the 6th century AD and at the time of the birth of Islam, a genetic group began to take shape that formed the genetic basis of the Druze communitys ancestors. According to this study, the Druze genome is largely similar to the genome of other Arab populations in the Middle East. They also found a few genetic elements in the Druze genome that originated from Europe, Central and South Asia (the Iran region) and Africa.

Our next step is to try to identify the genetic component of common diseases in this sector using the traditional family structure in a study that will allow genetic decoding of regular genetic diseases and provide data on diseases that have a genetic basis, such as cancer and diabetes. We are also planning similar studies in the future of the Muslim and Christian populations in Israel, Prof. Friedman concluded.

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An International Genetic Study Confirms the History of the Druze Community

TIME Health Obesity New Genes Mean the Future of Obesity Treatment Could Get Personal Getty Images Scientists have uncovered a trove of new genetic targets that could lead to better treatments for obesity

It took the genomes of nearly 340,000 people and more than 400 researchers in two dozen countries, but we now have the most comprehensive picture so far of the genetic contributors to obesity.

Two new papers in the journal Nature describe the results of two studies that connected the obesity-related factors of body mass index (the ratio between height and weight) and fat distribution to their potential genetic drivers. The studies did not isolate specific genesat least not yetbut identified areas in the human genome where people with different BMIs and different patterns of fat distribution varied in their genetic code. Those variants will lead scientists to the genes they code for, and eventually to how those genes work in contributing to obesity.

MORE: Healthy-Obesity Gene FoundBut Genes Arent Everything

I think we have so many more opportunities now to learn about the biology of obesity through genetic contributions to these traits, says Karen Mohlke, professor of genetics at University of North Carolina and the senior author of the report focusing on body fat distribution.

Those genetic clues may yield new weight-management treatments that are both more powerful and more personalized. What the data supports is the fact that there are a lot of different causes of obesity, says Dr. Elizabeth Speliotes, assistant professor of internal medicine and computational medicine and bioinformatics at the University of Michigan and senior author of the paper on body mass index. If youre hoping for one cause of obesity, thats not reality. What causes you to be obese is probably slightly different from what causes me to be obese.

Currently, however, all obesity is treated pretty much the same way. With the new knowledge gleaned from the genetics of whats driving different types of obesity, that may change.

MORE: Gym vs. Genes: How Exercise Trumps Obesity Genes

In the study involving factors contributing to BMI, Speliotes and her team discovered 97 genetic regions, or loci that account for nearly 3% of the variation among people on BMI. Of those, 56 are entirely new. Many of the regions are in areas that code for nervous system functions, or brain systems. Some arent so surprisingthey confirm previous studies that have implicated genetic regulators of areas that control appetite, for examplebut others were more unexpected. They involved regions responsible for learning, memory and even emotional regulation, hinting that some of weight and obesity may be tied to the addiction and reward pathways that help to reinforce behaviors like eating with feelings of pleasure and satisfaction. There were definitely a lot more loci involving the brain than I would have guessed, says Dr. Joel Hirschhorn, director of the center for basic and translational obesity research at Boston Childrens Hospital and Harvard Medical School and one of the co-authors. That makes obesity much more of a neurobehavioral disorder than just the fact that your fat cells are more efficient or less efficient.

MORE: Study Identifies Four New Genetic Markers For Severe Childhood Obesity

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New Genes Mean the Future of Obesity Treatment Could Get Personal

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Newswise ANN ARBOR, Mich. There are many reasons why people gain different amounts of weight and why fat becomes stored in different parts of their bodies. Now researchers are homing in on genetic reasons. Their findings, part of the largest genome-wide study to date, were published in two companion papers today in the journal Nature.

By analyzing genetic samples from more than 300,000 individuals to study obesity and body fat distribution, researchers in the international Genetic Investigation of Anthropometric Traits (GIANT) Consortium completed the largest study of genetic variation to date, and found over 140 locations across the genome that play roles in various obesity traits.

By applying novel computational methods to the genetic results, they discovered new biological pathways that are important in controlling body weight and fat distribution.

This work is the first step toward finding individual genes that play key roles in body shape and size. The proteins these genes help produce could become targets for future drug development.

Obesity is a global public health burden that affects millions of people. Yet, there are no long-term treatments.

Waist-to-hip ratios key for health risk One paper focused on where fat is stored in the body, one determinant of health risk. One of the observable traits linked to the genetic locations was waist-to-hip circumference ratio. People with waistlines larger than hip circumferences have more belly fat surrounding their abdominal organs. This makes them more likely to have metabolic conditions, such as type-2 diabetes, and cardiovascular problems than do people with body fat concentrated more in the hip area or distributed equally throughout the body.

We need to know these genetic locations because different fat depots pose different health risks, says Karen Mohlke, Ph.D., professor of genetics at the University of North Carolina School of Medicine and senior author of the paper that examined waist-to-hip ratio of fat distribution. If we can figure out which genes influence where fat is deposited, it could help us understand the biology that leads to various health conditions, such as insulin resistance/diabetes, metabolic syndrome, and heart disease.

The genetic locations associated with fat depots are associated with genes previously identified as being important for the creation of adipose tissue. Researchers also determined that 19 of the fat distribution genetic locations had a stronger effect in women; one had a stronger effect in men.

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Largest Ever Genome-Wide Study Strengthens Genetic Link to Obesity

Lets Play The Sims 3 100 baby/ Perfect Genetics Part 21: Baby #4
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10.02.2015 - (idw) Paul-Ehrlich-Institut - Bundesinstitut fr Impfstoffe und biomedizinische Arzneimittel

Therapeutic gene transfer is considered as a promising novel strategy to treat genetic disorders and cancer. So far, target cells are often isolated from patients for this purpose, and re-administered after gene transfer. In collaboration with colleagues from the Universities of Cologne and Zurich, researchers at the Paul-Ehrlich-Institut have succeeded in developing gene transfer vehicles that target the therapy relevant cell type directly in the organism. The resulting gene transfer occurs with an extremely high degree of selectivity. A report on the research results can be found in Nature Communications in its online edition of 10.02.2015. Vectors derived from adeno-associated viruses (AAV) were used as vehicles for targeted gene transfer by the research group of Professor Christian J. Buchholz, Principal Investigator at the LOEWE Centre for Cell and Gene Therapy at Frankfurt am Main and head of the Section Molecular Biotechnology and Gene Therapy of the President of the Paul-Ehrlich-Institut. AAV is a non-pathogenic parvovirus. The only gene therapy medicinal product authorised in Europe so far, is also based on AAV gene vectors and intended for the treatment of a rare metabolic disorder.

The strategy for the generation of the new precision gene vectors was developed and implemented jointly with Dr Hildegard Bning, head of the AAV Vector Development Research Group at the ZMMK (Zentrum fr Molekulare Medizin Kln, Center for Molecular Medicine Cologne) of the University of Cologne: Through exchange of two amino acids, AAV lost its ability to bind to its natural receptor and became thereby unable to penetrate its broad range of natural target cells. Novel target structures (DARPins, designed ankyrin repeat proteins) were then attached to the surface of the modified vector particles. These structures were developed at Zurich University. The structures can be selected in such a way that they mediate a selective binding of the DARPin-containing AAV vector particles to the therapy relevant cell type only. This is what enables the AAV vector to attach to and penetrate the desired target cell. The paper referenced here reports on the use of three different DARPins, which equipped AAV vectors either with a specificity for Her2/neu, a tumour marker in breast cancer, for EpCAM, an epithelial surface protein, or for a marker of particular blood cells (CD4 on the surface of lymphocytes with distinct immunological functions).

The desired goal of a cell type specific in vivo gene transfer was also achieved with the blood cell targeted vector: AAV transferred the gene only into lymphocytes present in spleen carrying the CD4 protein target structure.

The method developed by us jointly is a very promising tool both in fundamental research and for the targeted gene transfer in medicine, explained Dr Buchholz with regard to the current research results.

Original Publication

Mnch RC, Muth A, Muik A, Friedel T, Schmatz J, Dreier B, Trkola A, Plckthun A, Bning H, Buchholz CJ (2015): Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors. Nat Commun Feb 10 [Epub ahead of print]. http://www.nature.com/ncomms/2015/150210/ncomms7246/full/ncomms7246.html

The Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines in Langen near Frankfurt/Main, is a senior federal authority reporting to the Federal Ministry of Health (Bundesministerium fr Gesundheit, BMG). It is responsible for the research, assessment, and marketing authorisation of biomedicines for human use and veterinary vaccines. Its remit also includes the authorisation of clinical trials and pharmacovigilance, i.e. recording and evaluation of potential adverse effects. Other duties of the institute include official batch control, scientific advice and inspections. In-house experimental research in the field of biomedicines and life science form an indispensable basis for the varied and many tasks performed at the institute. The PEI, with its roughly 800 staff, also has advisory functions at a national level (federal government, federal states (Lnder)), and at an international level (World Health Organisation, European Medicines Agency, European Commission, Council of Europe etc.). Weitere Informationen:http://www.pei.de

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Precise gene transfer into therapy relevant cells after vector injection into blood

Portland, Oregon and Miami, Fla. (PRWEB) February 10, 2015

Global Stem Cells Group and the Regenerative Technology Alliance (RTA) have signed a memorandum of understanding to evaluate and promote stem cell training programs. RTA, a global provider of standards and certification for the emerging fields of regenerative medicine and science, will work with the Global Stem Cells Group to evaluate the regenerative medicine companys training programs and assess GSCGs participating physicians against the RTAs established international standards for the practice of regenerative and cell-based medicine.

Our new alliance with the RTA is a natural step toward establishing GSCGs recognition as a global leader in stem cell medicine, says Global Stem Cells Group CEO Benito Novas. This is a perfect fit for us, as Global Stem Cells Group shares the RTAs focus on high standards and transparency, especially when it comes to patient safety and advancing the field of stem cell medicine.

We are very pleased to have this alliance, says David Audley, General Secretary and Chair of the RTA. Our goal is to provide the highest level of transparency and oversight for the industry. Working with Global will allow us to have a direct and dramatic impact on physician training.

For more information, visit the Global Stem Cells Group website, email bnovas(at)stemcellsgroup(dot)com, or call 305-224-1858.

About Global Stem Cells Group:

Global Stem Cells Group, Inc. is the parent company of six wholly owned operating companies dedicated entirely to stem cell research, training, products, and solutions. Founded in 2012, the company combines dedicated researchers, physician and patient educators, and solution providers with the shared goal of meeting the growing worldwide need for leading edge stem cell treatments and solutions. With a singular focus on this exciting new area of medical research, Global Stem Cells Group and its subsidiaries are uniquely positioned to become global leaders in cellular medicine.

About the RTA

The Regenerative Technology Alliance (RTA) a global provider of standards and certification for the emerging fields of regenerative medicine and science, is a 501(c)3 and is supported by donations from individuals, corporations and foundations to help advance its critical mission of bringing peer oversight and transparency to the field of cell-based and regenerative medicine.

For more information visit the RTA website, email david(at)regen-tech(dot)org, or call 503-446-5039.

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Global Stem Cells Group Announces Alliance with Regenerative Technology

John Wyse, a shellfish farmer and father of three from Nanaimo, has not been able to work due to deteriorating health from a rare form of multiple sclerosis.

image credit: CHRIS BUSH/The News Bulletin

John Wyse, 40, a Nanaimo father of three, is in a race against the progression of his disease.

Wyse was diagnosed in 2010 with primary progressive multiple sclerosis and hopes to receive hematopoietic stem cell transplantation treatment at the Hassadah Medical Centre in Israel.

Multiple sclerosis affects the brain and spinal cord by causing inflammation that damages myelin the protective covering of the nerves and disrupts nerve impulses, giving rise to symptoms that include extreme fatigue, weakness, lack of coordination, impaired sensation, vision and bladder problems, cognitive impairment and mood changes.

What causes MS is unknown, but its thought to be an autoimmune disorder causing the bodys immune system to attack healthy tissue.

Patients suffer repeating cycles of advancing deterioration followed by periods of remission in all forms of MS except for the primary progressive variant of the disease, which progresses without remission and is the only form of MS for which there are no conventional drugs or treatments available.

Research into stem cell transplantation therapy is the latest avenue of hope for successful treatment and a possible cure. Clinics in Germany, Russia, India and Israel currently offer stem cell treatment and clinical trials are also being conducted in Canada, the U.S. and elsewhere.

Most clinical trials and some treatment clinics will not accept primary progressive MS patients.

Wyse, with his wife and three daughters, are trying to raise $158,200 to pay for his treatment in Israel, scheduled for April 2016, but the Hassadah Medical Centre places limits on how far Wyses condition can deteriorate before it will not accept him. Wyse, who now walks with a cane and hasnt been able to work for a year, figures he has little more than a year before hes no longer a treatment candidate.

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Hoping for a cure

A Conversation with Oxford #39;s Peter Donnelly: Genomics and Personalized Medicine
The TDWI/Hatch team had good fortune to attend the amazing Personalized Medicine World Conference put on by Tal and Gadi Behar at the Computer History Museum...

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Personalized Medicine: Right Drug, Right Dosage.
Dr. David Showers shares a case in which he was able to utilize the GenoPATH comprehensive medication assessment informed by genetics to change a patient #39;s m...

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Fundraiser held to support Lane Phillips healing from spinal cord injury
A Clarkston man is faced with a long recovery from serious injuries after an unexplained medical blackout in his home. A Clarkston man is faced with a long r...

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Fundraiser held to support Lane Phillips healing from spinal cord injury - Video

4 hours ago Inside the cell, calcium ions are released from a structure called the endoplasmic reticulum (ER). Forces applied to the bead cause ion channels in the ER to open mechanically (shown in red above), rather through biochemical signaling chemically (shown in green below). Credit: Jie Sun/UC San Diego

After using optical tweezers to squeeze a tiny bead attached to the outside of a human stem cell, researchers now know how mechanical forces can trigger a key signaling pathway in the cells.

The squeeze helps to release calcium ions stored inside the cells and opens up channels in the cell membrane that allow the ions to flow into the cells, according to the study led by University of California, San Diego bioengineer Yingxiao Wang.

Researchers have known that mechanical forces exerted on stem cells have a significant role to play in how the cells produce all kinds of tissuesfrom bone to bloodfrom scratch. But until now, it hasn't been clear how some of these forces translate into the signals that prod the stem cells into building new tissue.

The findings published in the journal eLife could help scientists learn more about "the functional mechanisms behind stem cell differentiation," said Wang, an associate professor of bioengineering. They may also guide researchers as they try to recreate these mechanisms in the lab, to coax stem cells into developing into tissues that could be used in transplants and other therapies.

"The mechanical environment around a stem cell helps govern a stem cell's fate," Wang explained. "Cells surrounded in stiff tissue such as the jaw, for example, have higher amounts of tension applied to them, and they can promote the production of harder tissues such as bone."

Stem cells living in tissue environments with less stiffness and tension, on the other hand, may produce softer material such as fat tissue.

Wang and his colleagues wanted to learn more about how these environmental forces are translated into the signals that stem cells use to differentiate into more specialized cells and tissues. In their experiment, they applied force to human mesenchymal stem cellsthe type of stem cells found in bone marrow that transform into bone, cartilage and fat.

The engineers used a highly focused laser beam to trap and manipulate a tiny bead attached to the cell membrane of a stem cell, creating an optical "tweezers" to apply force to the bead. The squeeze applied by the tweezers was extremely smallon the order of about 200 piconewtons. (Forces are measured in a unit called newtons; one newton is about the weight of an apple held to the Earth by gravity, and one piconewton is equivalent to one-trillionth of a newton.)

When there were no calcium ions circulating outside the cell, this force helped to release calcium ions from a structure inside the cell called the endoplasmic reticulum. The release is aided by the cell's inner structural proteins called the cytoskeleton, along with contracting protein machinery called actomyosin. When the force triggered the movement of calcium ions into the cell from its extracellular environment, only the cytoskeleton was involved, the researchers noted.

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Engineers put the 'squeeze' on human stem cells

Children with aggressive leukaemia experiencing remission with T cell therapy
Visit http://www.ecancer.org for more. Dr Grupp (The Children #39;s Hospital of Philadelphia, Philadelphia, USA) talks to ecancertv at ASH 2014 about the use of T cell therapy for children with...

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DUBLIN, Feb .10, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/7zf9mz/cell_therapy) has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

The cell-based markets was analyzed for 2014, and projected to 2024.The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 294 of these are profiled in part II of the report along with tabulation of 285 alliances. Of these companies, 160 are involved in stem cells. Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 61 Tables and 16 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

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Cell Therapy Report 2014-2020 - Technologies, Markets and Companies

University of North Carolina scientists have created a new research tool, based on the fruit fly, to help crack the histone code. This research tool can be used to better understand the function of histone proteins, which play critical roles in the regulation of gene expression in animals and plants.

This work, published in the journal Developmental Cell, opens the door to experiments that are expected to uncover new biology important for a host of conditions, such as neurological diseases, diabetes, obesity, and especially cancer, which has become a hotbed of epigenetic research.

"People think cancer is a disease of uncontrolled proliferation, but that's just one aspect of it," said Robert Duronio, PhD, professor of biology and genetics and co-senior author. "Cancer is actually a disease of development in which the cells don't maintain their proper functions; they don't do what they're supposed to be doing." Somehow, the gene regulation responsible for proper cell development goes awry.

One aspect of gene regulation involves enzymes placing chemical tags or modifications on histone proteins -- which control a cell's access to the DNA sequences that make up a gene. Properly regulated access allows cells to develop, function, and proliferate normally. The chemical modification of histones is thought to be a form of epigenetic information -- information separate from our DNA -- that controls gene regulation. This idea is based on the study of the enzymes that chemically modify histones. However, there is a flaw in this argument.

"In complex organisms, such as fruit flies, mice, and humans, scientists have only been able to infer how these enzymes mechanistically accomplish their tasks," said Daniel McKay, PhD, assistant professor of genetics and biology and first author of the paper. "It's been technically impossible to directly study the role of histone modifications. Now, through our collaboration between UNC biologists, we've been able to develop a tool in fruit flies to directly test the function of histones independently of the enzymes that modify them."

This is crucial because therapies, such as cancer drugs, can target histones. With this new research tool, scientists will be able to better study thousands of enzyme-histone interactions important for human health.

"If you think of the genome as a recipe book, then you could say we've made it possible to know that there are hidden ingredients that help explain how specific recipes turn out correctly or not," said Greg Matera, PhD, professor of biology and genetics and co-senior author of the paper. "That's the first step in scientific discovery -- knowing that there are things we need to look for and then searching for them."

Beyond Yeast

Before now, a lot of this epigenetic research had been done in yeast -- single cell organisms that also use enzymes to lay chemical tags on histone proteins. This work has yielded many interesting findings and has led to the development of therapeutics. But some of this work has led to an oversimplification of human biology, leaving many questions about human health unanswered.

For instance, in complex organisms, enzymes in cells typically do more than one thing. One likely reason for this is that animals undergo cellular differentiation; human life begins as a single cell that differentiates into the various cell types needed for different organs, body parts, blood, the immune system, etc. This differentiation has to be maintained throughout life.

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Epigenetic breakthrough: A first of its kind tool to study the histone code

Light-activated genes might be precisely controlled and targeted

By Ken Kingery

Duke University researchers have devised a method to activate genes in any specific location or pattern in a lab dish with the flip of a light switch by crossing a bacteriums viral defense system with a flowers response to sunlight.

With the ability to use light to activate genes in specific locations, researchers can better study genes functions, create complex systems for growing tissue, and perhaps eventually realize science-fiction-like healing technologies.

The study was led by Charles Gersbach, assistant professor of biomedical engineering at Duke University, and published on February 9 in Nature Chemical Biology.

Researchers demonstrate their new technique to control genes by shining light through a Duke D stencil to turn on fluorescent genes in cells.

The new technique targets specific genes using an emerging genetic engineering system called CRISPR/Cas9. Discovered as the system bacteria use to identify viral invaders and slice up their DNA, the system was co-opted by researchers to precisely target specific genetic sequences.

The Duke scientists then turned to another branch of the evolutionary tree to make the system light-activated.

In many plants, two proteins lock together in the presence of light, allowing plants to sense the length of day which determines biological functions like flowering. By attaching the CRISPR/Cas9 system to one of these proteins and gene-activating proteins to the other, the team was able to turn several different genes on or off just by shining blue light on the cells.

Charles Gersbach

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Lighting Up the Duke 'D' With Genes

Members of England's Parliament passed legislation on Feb. 3 that allows the use of DNA from a third-party female donor to be used in a human embryo -- a new move toward eliminating genetic disease.

The technique could help babies with mitochondrial disease, which affects one in every 6,500 babies and can be fatal. Mitochondria, found in nearly all of the body's cells, converts food into usable energy and contains DNA that does not affect other personal traits (such as appearance). When defective, it can lead to brain damage, heart failure, blindness and muscle wasting.

The process would replace the harmful material in a woman's egg prior to conception, mixing the DNA of the two parents with a donor woman's healthy mitochondria.

This procedure, however, raises a number of ethical issues and objections, as it involves experimentation with human reproduction and requires in vitro fertilization, the church's "default" argument against the technique, wrote Jack Mahoney for The Tablet.

But the church also once opposed organ transplants for requiring "self-mutilation," Mahoney noted. "Few people would now accept that extremely partial analysis as an adequate description of what many rightly view as an act of human solidarity," he wrote.

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The distinction between genetic transplants and a "genetic apocalypse" likens in positive or negative genetic medicine. "It need not be the case that the wish to prevent an individual, or even the human gene pool, from suffering a particular genetic malady will inevitably usher humanity into Aldous Huxley's Brave New World," Mahoney wrote, referring to a 1932 novel about reproductive technology.

Negative, or preventive, genetic medicine aims to eliminate deficiencies or diseases in an individual or possibly for generations to come. Positive genetic medicine, or genetic enhancement, aspires to improve individuals by adding genes based on preferences, making the individual more personally or socially advantageous. When geared for the individual's sake, it is "somatic therapy," whereas "germ line therapy" refers to the introduction of genetic changes in the reproductive system that will eventually continue through succeeding generations.

But the uneasiness around substituting various genes, Mahoney said, implies "the view that humans are simply the product of their genes, now including someone else's, and that their personality and behaviour are determined by their genetic make-up, leaving little, if any, room for personal freedom of choice and self-determination on the part of the individual."

Consider how external factors -- environmental, economic, and the unconscious -- affect human behavior. "Being predisposed is not the same as being predetermined," Mahoney wrote.

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Another look at the potential for three-parent babies

Precision medicine seems to be the new hot topic in the research world. President Obama spoke about precision medicine in his State of the Union speech on January 20, his budget released today requests $215M for precision medicine, and NIH just announced plans for a study of a million or more volunteers to explore precision medicine. What precisely is it? The White House website has a useful definition: getting the right treatment at the right time to the right person. The President, in an event devoted to precision medicine in the East Room last Friday [January 30, see video, below], told the story of ivacaftor, a drug that effectively treats the underlying causenot the symptomsof cystic fibrosis, but works in only 4% of patients who have a specific mutation in the gene causing this disease.

Most of the conversation about precision medicine focuses on cancer. Because cancer is a disease of genetic mutations leading to unregulated cell division, defining the precise mutations in the affected tissue have already led to breakthrough treatments for both blood and solid tissue cancers. In fact, the same mutation can occur in different parts of the body, so cancer is increasingly diagnosed in terms of its genetic and molecular signature rather than the tissue of origin. Part of the proposed precision medicine plan will involve scaling up efforts at the National Cancer Institute to find these mutations and to develop drugs or biologics as treatments.

What does precision medicine mean for mental health? Our version is the Research Domain Criteria (RDoC) project, which aims to develop more precise diagnostic categories based on biological, psychological, and socio-cultural variables. It is certainly possible that we may find specific mutations in relevant brain circuits that explain some cases of schizophrenia, bipolar disorder, or autism, just as mutations in the tumor explain cancer. NIMH has supported research on inherited genetic risk for several years; a new initiative on another class of mutations, somatic mosaicism (the term for mutations that develop after fertilization), will launch this year. But more likely, precision medicine for mental disorders will not come from a single genomic glitch. Rather, like many other areas of medicine, many genes each contribute only a small amount of vulnerability as part of an overall risk profile that includes life experiences, neurodevelopment, and social and cultural factors. RDoC assumes that we will need many kinds of data to reach precision, more like triangulating to find your position on a map. These data will draw from many sources, including symptoms, genotype, physiology, cognitive assessment, family dynamics, environmental exposures, and cultural background.

I know that RDoC sounds more complex than the cancer version of precision medicine, but that is the nature of the problem. For now, we need to embrace the complexity to identify the simpler, actionable categories that can be used in the clinic. The rationale for this approach is no different from what the President talked about for cancer or cystic fibrosisgetting the right treatment at the right time to the right person.

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Precision Medicine for Mental Disorders

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Virology 2015 Lecture #3: Genomes and Genetics
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Medical Genetics- Autosomal Dominant Disorders [Video Lecture]
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By RTT News, February 10, 2015, 04:32:00 PM EDT

(RTTNews.com) - Biotechnology company Seattle Genetics Inc ( SGEN ) on Tuesday reported fourth-quarter net loss of $26.7 million or $0.22 per share compared with a loss of $15.7 million or $0.13 per share last year.

Revenues for the quarter were $74.3 million compared with $67.4 million in the prior year.

Analysts polled by Thomson Reuters estimated a loss of $0.25 per share on revenues of $69.1 million for the quarter. Analysts' estimates typically exclude special items.

Total expenses for the quarter rose to $102 million from $83 million last year, due mainly to higher research costs.

For the full year 2015 , the company expects revenues from sales of key product ADCETRIS of $200 million to $210 million and revenues from collaboration and license agreements of $60 million to $70 million.

Analysts expect revenues of $332.9 million for 2015.

For comments and feedback: contact editorial@rttnews.com

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Seattle Genetics Loss Narrower Than Estimates

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Cell Therapy, which is based in the Welsh capital Cardiff, says the medicine has the potential to reduce scarring of the heart muscle caused by a heart attack or failure.

Chief executive Ajan Reginald, who was previously at Roche, said crowd funding was a quick way to raise money for final stage trials or commercial launches.

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"It was very fast and very efficient," he said. "We have spent five per cent of our time on fundraising, which enables me to spend 95 per cent of my time on the business."

The company's founder Martin Evans shared the 2007 Nobel Prize for medicine for groundbreaking stem cell research.

Cell Therapy used website Crowdcube to raise nearly three times its original target from more than 300 investors.

Mr Reginald said the backers included investment bankers, hedge fund employees and scientists.

"Crowd funding allows investors to look in detail at a company in their own time," he said, adding that some 10,000 investors had seen the pitch.

The company plans to publish data from clinical trials of the drug, called Heartcel, next month, before final stage trials with a view to a launch in 2016.

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Biotech firm Cell Therapy claims crowdfunding record with heart drug

Biotechs may be flush with cash, thanks to the ol bullish IPO market and an uptick in venture funding. But startups remainon the lookout for alternative funding models with crowdsourcing front and center.

This makes British biotech startup Cell Therapyparticularly interesting,itjustraised 689,246 or a bit over$1 million to launch a stem cell therapy for heart failure. This is one of the highest life sciences-related crowdfunding efforts topped only by Scanadu, whose handheld consumer diagnostic tool raised $1.6 million in Indiegogo.

Cell Therapy, which was founded by 2007 Nobel Prize winner Martin Evans, raised the funding on thesite Crowdcube exceeding its goal of 250,000 with backing from nearly 300 investors. It ceded a mere 0.39% in equity to the backers thatinclude investment bankers, hedge fund employees and scientists, CEO Ajan Reginald said.

It was very fast and very efficient, Reginaldtold Reuters. We have spent 5 percent of our time on fundraising, which enables me to spend 95 percent of my time on the business.

Crowdfunding is increasingly becoming an option for early stage biotechs that want to sidestep the traditional venture-backed approach. On one hand, its a relatively simple means to raise a large amount of seed capital but on the other, there are many more (potentially irate) investors to answer to when a companys in its nascence.

New York-based Poliwoggs entire premise is on bringing crowdfunding to healthcare with aims to help companies raise fundsfrom accredited investors beyond the seed stage, with rounds ranging from $2 million to $10 million mark.Notably, ithas its own regenerative medicine fund.

Part of the idea here is that people want to invest in the things they care about, but they havent always had the opportunity to invest in them, CEO Greg Simon told MedCity News.Were giving people the opportunity to put their money where their passion is.

Thats all fine and good to have a passion for a cause, but the traditional accredited investor whos enmeshed in a crowdfunding effort may still not understand the intricacies of what it takes to get results or a return in a tricky field like regenerative medicine.

John Carroll over atFierce Biotechopined that crowdfunding wont make a significant dent in the approach to life sciences crowdfunding. Stem cell therapy, after all, generated tons of media pomp and flair a decade ago, but has yet to deliver on many of its curative promises from back then. VCs are often burnt and reticent, and investors on crowdfunding sites will likely be, as well. Carroll says:

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Cell Therapy may have just raised $1M, but will crowdfunding have a lasting place in biotech?