PUBLIC RELEASE DATE:

13-Dec-2013

Contact: Emma Rayner emma.rayner@nottingham.ac.uk 44-011-595-15793 University of Nottingham

A research team from The University of Nottingham has helped uncover a second rare genetic mutation which strongly increases the risk of Alzheimer's disease in later life.

In an international collaboration, the University's Translational Cell Sciences Human Genetics research group has pinpointed a rare coding variation in the Phospholipase D3 (PLD3) gene which is more common in people with late-onset Alzheimer's than non-sufferers.

The discovery is an important milestone on the road to early diagnosis of the disease and eventual improved treatment. Having surveyed the human genome for common variants associated with Alzheimer's, geneticists are now turning the spotlight on rare mutations which may be even stronger risk factors.

More than 820,000 people in the UK have dementia and the number is rising as the population ages. The condition, of which Alzheimer's disease is the predominant cause, costs the UK economy 23 billion per year, much more than other diseases like cancer and heart disease.

Nottingham's genetic experts have been working with long-term partners from Washington University, St Louis, USA and University College, London, to carry out next-generation whole exome sequencing on families where Alzheimer's affects several members.

Earlier this year the collaboration uncovered the first ever rare genetic mutation implicated in disease risk, linking the TREM2 gene to a higher risk of Alzheimer's (published in the New England Journal of Medicine). Now, in a new study published today in the international journal, Nature, the team reveal that after analysis of the genes of around 2,000 people with Alzheimer's, a second genetic variation has been found, in the PLD3 gene.

PLD3 influences processing of amyloid precursor protein which results in the generation of the characteristic amyloid plaques seen in AD brain tissue, suggesting that it may be a potential therapeutic target.

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New gene discovery sheds more light on Alzheimer's risk

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Rajeev Saini MD, recently passed the American Board of Obesity Medicine Certification Exam for Obesity Medicine Physicians and became certified in the subspecialty of obesity medicine.

Physicians certified in obesity medicine are able to positively impact all systems of the body by incorporating weight management into traditional models of medical practice. Specifically, this subspecialty requires competency in and a thorough understanding of the treatment of obesity and the genetic, biologic, environmental, social and behavioral factors that contribute to obesity.

Rajeev Saini

Dr. Saini was among only 191 physicians nationally who successfully completed this years exam.

According to the Centers for Disease Control and Prevention, more than one-third of adults and 17 percent of children and adolescents in this country are obese. Obesity-related conditions include heart disease, stroke, type 2 diabetes and certain types of cancer. In 2008, medical costs associated with obesity were estimated at $147 billion.

Supervision from a health care provider is necessary to detect and treat weight-related medical conditions, Dr. Saini said. A healthcare program supervised by a physician, certified in obesity medicine offers a comprehensive and effective approach to maximizing overall health and reversing obesity-related co-morbidities.

In New York State, the prevalence of adult obesity is about 25 percent. said Dr. Saini. I am interested in being part of the solution to this medical epidemic by locally helping patients affected by obesity who seek access to non-surgical treatment options. It is amazing to see as people lose weight, we are able to cut down or even stop their medications for various medical conditions like diabetes, hypertension, hyperlipidemia, etc.

Patients and their health care providers need a variety of options for obesity treatments, since no single treatment approach will work for every individual. The American Society of Bariatric Physicians provides guidance to its members through a set of obesity medicine guidelines, which outline four medical weight-loss methods: dietary modification, exercise prescription, behavior modification and, when appropriate, medication.

Dr. Saini has been in practice for the last 17 years. He is board certified in Internal Medicine and has offices in Fulton and Baldwinsville. He went to medical school at AIIMS, India and did his residency in Internal Medicine at Upstate Medical University.

Since its founding in January 2012, ABOM has sought to further the accreditation of a sub-specialization of medical practice: obesity medicine.

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Local Physician Earns Certification In Obesity Medicine

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PUBLIC RELEASE DATE:

13-Dec-2013

Contact: Beth Ruedi eruedi@genetics-gsa.org 301-634-7371 Genetics Society of America

BETHESDA, MD December 13, 2013 The Genetics Society of America (GSA) is pleased to announce the selection of eleven early career researchers five graduate students and six postdoctoral researchers as recipients of a spring 2014 DeLill Nasser Award for Professional Development in Genetics. The award is a $1,000 travel grant for each researcher to attend any national or international meeting, conference or laboratory course that will enhance his or her career.

"GSA is always honored to present the DeLill Nasser Awards because they are about promoting the future of our discipline," said Adam Fagen, PhD, Executive Director of GSA. "Attending scientific conferences and courses is an essential element of practicing science, and we are glad to play a role in fostering the professional development of some of our most promising early career members."

The DeLill Nasser Award was established by GSA in 2001 to honor its namesake, DeLill Nasser (1929-2000), a long-time GSA member who provided critical support to many early career researchers during her 22 years as program director in eukaryotic genetics at the National Science Foundation. Since the formation of this award, over 100 graduate students and postdocs have received funding for travel to further their career goals and enhance their education. The program is supported by GSA, and with charitable donations from members of the genetics community.

The eleven recipients of the spring 2014 DeLill Nasser Awards, their institutions, the conference or course each intends to attend, and a brief summary of their research is listed below.

Graduate Students:

Yang Cao University of WisconsinMadison, Madison, WI GSA 55th Annual Drosophila Research Conference March 26-30, 2014, San Diego, CA "I am investigating two fundamental neurobiological processes, synaptic development and neuroprotection, using Drosophila as an animal model."

Huan Chen Columbia University Medical Center, New York, NY Mechanisms of Recombination Meeting May 19-23, 2014, Alicante, SPAIN "I am studying the molecular mechanism of DNA double-strand break repair and genome integrity in eukaryotes."

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Genetics Society of America announces recipients of spring 2014 DeLill Nasser Award

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Newswise BETHESDA, MD December 13, 2013 The Genetics Society of America (GSA) is pleased to announce the selection of eleven early career researchers five graduate students and six postdoctoral researchers as recipients of a spring 2014 DeLill Nasser Award for Professional Development in Genetics. The award is a $1,000 travel grant for each researcher to attend any national or international meeting, conference or laboratory course that will enhance his or her career.

GSA is always honored to present the DeLill Nasser Awards because they are about promoting the future of our discipline, said Adam Fagen, PhD, Executive Director of GSA. Attending scientific conferences and courses is an essential element of practicing science, and we are glad to play a role in fostering the professional development of some of our most promising early career members.

The DeLill Nasser Award was established by GSA in 2001 to honor its namesake, DeLill Nasser (19292000), a long-time GSA member who provided critical support to many early career researchers during her 22 years as program director in eukaryotic genetics at the National Science Foundation. Since the formation of this award, over 100 graduate students and postdocs have received funding for travel to further their career goals and enhance their education. The program is supported by GSA, and with charitable donations from members of the genetics community.

The eleven recipients of the spring 2014 DeLill Nasser Awards, their institutions, the conference or course each intends to attend, and a brief summary of their research is listed below.

PLEASE NOTE: a full media release complete with pictures is available here.

Graduate Students:

Yang Cao University of WisconsinMadison, Madison, WI GSA 55th Annual Drosophila Research Conference March 2630, 2014, San Diego, CA I am investigating two fundamental neurobiological processes, synaptic development and neuroprotection, using Drosophila as an animal model.

Huan Chen Columbia University Medical Center, New York, NY Mechanisms of Recombination Meeting May 1923, 2014, Alicante, SPAIN I am studying the molecular mechanism of DNA double-strand break repair and genome integrity in eukaryotes.

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Genetics Society of America Announces Recipients of Spring 2014 DeLill Nasser Award for Professional Development

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In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients blood cells into soldiers that seek and destroy cancer.

A few patients with one type of leukemia were given this one-time, experimental therapy several years ago and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.

Its really exciting, said Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. You can take a cell that belongs to a patient and engineer it to be an attack cell.

In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia, or ALL, had a complete remission, meaning no cancer could be found after treatment, although a few have relapsed since then.

These were gravely ill patients out of options. Some had tried multiple bone marrow transplants and up to 10 types of chemotherapy or other treatments.

Cancer was so advanced in 8-year-old Emily Whitehead of Philipsburg, Pa., that doctors said her major organs would fail within days. She was the first child given the gene therapy and shows no sign of cancer today, nearly two years later.

Emily Whitehead, 8, 20 months after T Cell Therapy treatment in Philadelphia.Photo: AP

Results on other patients with myeloma, lymphoma and chronic lymphocytic leukemia, or CLL, will be reported at the hematology groups conference that starts Saturday in New Orleans.

Doctors say this has the potential to become the first gene therapy approved in the United States and the first for cancer worldwide. Only one gene therapy is approved in Europe, for a rare metabolic disease.

The treatment involves filtering patients blood to remove millions of white blood cells called T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the patient in infusions over three days.

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Leukemia patients in complete remission after gene therapy

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Newswise Scientists from UCLAs Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research are bringing stem cell science funded by the California Institute of Regenerative Medicine (CIRM), the state stem cell agency, directly to patients in two exciting new clinical trials scheduled to begin in early 2014. The recipients of the Disease Team Therapy Development III awards were Dr. Dennis Slamon and Dr. Zev Wainberg, whose phase I clinical trial will test a new drug that targets cancer stem cells and has been approved to begin enrolling patients in the US and Canada, and Dr. Donald Kohn, whose first-in-human trial is on stem cell gene therapy for sickle cell disease (SCD).

The announcement of the new awards came on December 12, 2013 at the meeting of the CIRM Independent Citizens Oversight Committee (ICOC) at the Luxe Hotel in Los Angeles. Dr. Owen Witte, Director of the UCLA Broad Stem Cell Research Center, highlighted that the The CIRM support demonstrates that our multidisciplinary Center is at the forefront of translating basic scientific research to new drug and cellular therapies that will revolutionize medicine.

Targeting solid tumor stem cells The Disease Team III grant to Dr. Dennis Slamon and Dr. Zev Wainberg and their US-Canadian collaborative team will support the first in human clinical trial scheduled to open in early 2014. The project builds on Dr. Slamons previous work partially funded by CIRM to develop a drug that targets tumor initiating cells with UCLAs Dr. Zev Wainberg, assistant professor of hematology/oncology and Dr. Tak Mak, director, Campbell Family Institute of the University Health Network in Toronto, Canada. Dr. Slamon, renowned for his research that led to the development of Herceptin, the first FDA-approved targeted therapy for breast cancer, is the director of clinical and translational research at the UCLA Jonsson Comprehensive Cancer Center, and professor, chief and executive vice chair for research in the division of hematology/oncology.

With investigational new drug approval from the Food and Drug Administration (FDA) and Health Canada, the Canadian governments therapeutic regulatory agency, this trial is an international effort to bring leading-edge stem cell science to patients.

We are delighted to receive this CIRM grant that will drive our translational research from the laboratory to the clinic, Slamon said, and allow us to test our targeted drug in a phase I clinical trial.

The trial is based on the evidence built over the last decade for what has become known as the cancer stem cell hypothesis. According to this hypothesis, cancer stem cells are the main drivers of tumor growth and are also resistant to standard cancer treatments. One view is that cancer stem cells inhabit a niche that prevents cancer drugs from reaching them. Another view is that tumors can become resistant to therapy by a process called cell fate decision, by which some tumor cells are killed by therapy and others become cancer stem cells. These cancer stem cells are believed to be capable of self-renewal and repopulation of tumor cells, resulting in the recurrence of cancer.

The target of the new drug is an enzyme in cancer stem cells and tumor cells called Polo-like kinase 4, which was selected because blocking it negatively affects cell fate decisions associated with cancer stem cell renewal and tumor cell growth, thus stopping tumor growth.

This potential anti-cancer drug is now ready to be tested in humans for the first time. Our goal is to test this novel agent in patients in order to establish safety and then to proceed quickly to rapid clinical development. We are excited to continue this academic collaboration with our Canadian colleagues to test this drug in humans for the first time, said Wainberg. Drs. Slamon, Wainberg, Mak and colleagues will also look for biological indications, called biomarkers, that researchers can use to tell if and how the drug is working.

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UCLA Scientists Taking Stem Cell Research to Patients

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Durham, NC (PRWEB) December 09, 2013

Generating new cardiac muscle from human embryonic stem cells (hESCs) and/or induced pluripotent stem cells (iPSC) could fulfill the demand for therapeutic applications and drug testing. The production of a similar population of these cells remains a major limitation, but in a study just published in STEM CELLS Translational Medicine, researchers now believe they have found a way to do this.

By combining small molecules and growth factors, the international research team led by investigators at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai developed a two-step system that caused stem cells to differentiate into ventricular heart muscle cells from hESCs and iPSCs. The process resulted in high efficiency and reproducibility, in a manner that mimicked the developmental steps of normal cardiovascular development.

These chemically induced, ventricular-like cardiomyocytes (termed ciVCMs) exhibited the expected cardiac electrophysiological and calcium handling properties as well as the appropriate heart rate responses, said lead investigator Ioannis Karakikes, Ph.D., of the Stanford University School Of Medicine, Cardiovascular Institute. Other members of the team included scientists from the Icahn School of Medicine at Mount Sinai, New York, and the Stem Cell & Regenerative Medicine Consortium at the University of Hong Kong.

In addition, using an integrated approach involving computational and experimental systems, the researchers demonstrated that using molecules to modulate the Wnt pathway, which passes signals from cell to cell, plays a key role in whether a cell evolves into an atrial or ventricular muscle cell.

The further clarification of the molecular mechanism(s) that underlie this kind of subtype specification is essential to improving our understanding of cardiovascular development. We may be able to regulate the commitment, proliferation and differentiation of pluripotent stem cells into heart muscle cells and then harness them for therapeutic purposes, Dr. Karakikes said.

"Most cases of heart failure are related to a deficiency of heart muscle cells in the lower chambers of the heart, said said Anthony Atala, MD, editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. An efficient, cost-effective and reproducible system for generating ventricular cardiomyocytes would be a valuable resource for cell therapies as well as drug screening.

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The full article, Small Molecule-Mediated Directed Differentiation of Human Embryonic Stem Cells Toward Ventricular Cardiomyocytes, can be accessed at http://www.stemcellstm.com.

About STEM CELLS Translational Medicine: STEM CELLS TRANSLATIONAL MEDICINE (SCTM), published by AlphaMed Press, is a monthly peer-reviewed publication dedicated to significantly advancing the clinical utilization of stem cell molecular and cellular biology. By bridging stem cell research and clinical trials, SCTM will help move applications of these critical investigations closer to accepted best practices.

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More Efficient Way to Grow Heart Muscle from Stem Cells Could Yield New Regenerative Therapies

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NASA and the Center for the Advancement of Science in Space (CASIS) are enabling research aboard the International Space Station that could lead to new stem cell-based therapies for medical conditions faced on Earth and in space.

Scientists will take advantage of the space station's microgravity environment to study the properties of non-embryonic stem cells.

NASA is interested in space-based cell research because it is seeking ways to combat the negative health effects astronauts face in microgravity, including bone loss and muscle atrophy. Mitigation techniques are necessary to allow humans to push the boundaries of space exploration far into the solar system. This knowledge could help people on Earth, particularly the elderly, who are afflicted with similar conditions.

Two stem cell investigations scheduled to fly to the space station next year were highlighted Friday, Dec. 6, at the World Stem Cell Summit in San Diego. Lee Hood, a member of the CASIS Board of Directors, moderated a panel session in which scientists Mary Kearns-Jonker of Loma Linda University in California and Roland Kaunas of Texas A&M University discussed their planned research, which will gauge the impact of microgravity on fundamental stem cell properties.

Kearns-Jonker's research will study the aging of neonatal and adult cardiac stem cells in microgravity with the ultimate goal of improving cardiac cell therapy. Kaunas is a part of a team of researchers developing a system for co-culturing and analyzing stem cells mixed with bone tumor cells in microgravity. This system will allow researchers to identify potential molecular targets for drugs specific to certain types of cancer.

Stem cells are cells that have not yet become specialized in their functions. They display a remarkable ability to give rise to a spectrum of cell types and ensure life-long tissue rejuvenation and regeneration. Experiments on Earth and in space have shown that microgravity induces changes in the way stem cells grow, divide and specialize. Stem cell biology in microgravity could inform fields ranging from discovery science to tissue engineering to regenerative medicine.

NASA selected CASIS to maximize use of the International Space Station's U.S. National Laboratory through 2020. CASIS is dedicated to supporting and accelerating innovations and new discoveries that will enhance the health and wellbeing of people and our planet.

For more information about the International Space Station, visit:

http://www.nasa.gov/station

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NASA, CASIS Make Space Station Accessible for Stem Cell Research

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New research suggests that outcomes for patients who have undergone stem cell transplants from unrelated or mismatched donors could be improved with the use of a drug called bortezomib, also known as velcade. This is according to a study presented at the annual meeting of the American Society of Hematology.

Stem cell transplants are treatments carried out in an attempt to cure some cancers affecting the body's bone marrow, such as leukemia, lymphoma and myeloma.

The treatment involves very high doses of chemotherapy (myeloablation) or whole body radiotherapy to clear a person's bone marrow and immune system of cancerous cells.

After this process, the killed cells are replaced with healthy stem cells through a drip that flows into a vein. These stem cells can be from the patient's own body or from a donor - preferably a sibling.

According to researchers from the Dana-Farber Cancer Institute who conducted the study, stem cells from unrelated or mismatched donors are likely to lead to worse patient outcomes following transplantation.

These patients tend to have a higher mortality rate as a result of the treatment and are more likely to experience graft-versus-host-disease (GVHD). This is a disease in which the transplanted cells attack the immune system of the recipient.

According to the researchers, recipients of mismatched donor transplants have a severe GVHD rate of 37%, a 1-year treatment-related mortality rate of 45%, and a 1-year overall survival rate of 43%.

Recipients of unrelated donor transplants have a severe GVHD rate of 28%, a 1-year treatment-related mortality rate of 36%, and a 1-year overall survival rate of 52%.

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Stem cell transplantation outcomes 'improved with new drug regime'

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Dec. 9, 2013 at 10:17 AM ET

Two men who had hoped they might be cured of an HIV infection after getting bone marrow transplants for cancer got some bad news, doctors said Monday. The virus has come back.

The intense and life-threatening treatments for cancer appeared to have wiped the virus out, and the two men took a chance and, earlier this year, stopped taking the HIV drugs that were keeping the virus under control.

At first, no signs of the virus could be found. But their doctors, cautious after decades of fighting a tricky virus, didnt declare a cure.

Its disappointing, said Dr. Daniel Kuritzkes of Brigham and Womens Hospital in Boston, who worked with Dr. Timothy Henrich to treat and study the two men.

But its still taught us a great deal.

The case of the two men shows that even if you make HIV seemingly disappear, it can be hiding out in the body and can re-activate. It might be somewhere other than in blood cells, Henrich said. Other scientists suspect HIV might be able to hole up in organs or inside the intestines.

Through this research we have discovered the HIV reservoir is deeper and more persistent than previously known and that our current standards of probing for HIV may not be sufficient to inform us if long-term HIV remission is possible if antiretroviral therapy is stopped, Henrich said.

Both patients have resumed therapy and are currently doing well. Neither man wants to be named.

Henrich, Kuritzkes and colleagues had actively looked for HIV patients with leukemia or lymphoma who had received bone marrow stem cell transplants.

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AIDS virus comes back in men who hoped for cure

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Newswise HOUSTON (Dec. 10, 2013) A first-of-its-kind clinical trial studying two forms of stem cell treatments for children with cerebral palsy (CP) has begun at The University of Texas Health Science Center at Houston (UTHealth) Medical School.

The double-blinded, placebo-controlled studys purpose includes comparing the safety and effectiveness of banked cord blood to bone marrow stem cells. It is led by Charles S. Cox, Jr., M.D., the Childrens Fund, Inc. Distinguished Professor of Pediatric Surgery at the UTHealth Medical School and director of the Pediatric Trauma Program at Childrens Memorial Hermann Hospital. Co-principal investigator is Sean I. Savitz, M.D., professor and the Frank M. Yatsu, M.D., Chair in Neurology in the UTHealth Department of Neurology.

The study builds on Cox extensive research studying stem cell therapy for children and adults who have been admitted to Childrens Memorial Hermann and Memorial Hermann-Texas Medical Center after suffering a traumatic brain injury (TBI). Prior research, published in the March 2010 issue of Neurosurgery, showed that stem cells derived from a patients own bone marrow were safely used in pediatric patients with TBI. Cox is also studying cord blood stem cell treatment for TBI in a separate clinical trial.

A total of 30 children between the ages of 2 and 10 who have CP will be enrolled: 15 who have their own cord blood banked at Cord Blood Registry (CBR) and 15 without banked cord blood. Five in each group will be randomized to a placebo control group. Families must be able to travel to Houston for the treatment and follow-up visits at six, 12 and 24 months.

Parents will not be told if their child received stem cells or a placebo until the 12-month follow-up exam. At that time, parents whose children received the placebo may elect to have their child receive the stem cell treatment through bone marrow harvest or cord blood banked with CBR.

Collaborators for the study include CBR, Lets Cure CP, TIRR Foundation and Childrens Memorial Hermann Hospital. The study has been approved by the U.S. Food and Drug Administration.

Cerebral palsy is a group of disorders that affects the ability to move and maintain balance and posture, according to the Centers for Disease Control. It is caused by abnormal brain development or damage to the developing brain, which affects a persons control over muscles. Treatment includes medications, braces and physical, occupational and speech therapy.

For a list of inclusion and exclusion criteria for the trial, go to http://www.clinicaltrials.gov. For more information, call the toll-free number, 855-566-6273.

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UTHealth Researchers Study Stem Cell Treatments for Children with CP

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PUBLIC RELEASE DATE:

9-Dec-2013

Contact: Jim Ritter jritter@lumc.edu 708-216-2445 Loyola University Health System

MAYWOOD, Il. - Donated umbilical cord blood contains stem cells that can save the lives of patients with leukemia, lymphoma and other blood cancers.

Now a study lead by a Loyola University Medical Center oncologist has found that growing cord blood stem cells in a laboratory before transplanting them into patients significantly improves survival.

The cell-expansion technology potentially could boost the number of patients who could benefit from life-saving transplants of stem cells derived from umbilical cord blood, said Patrick Stiff, MD, lead author of the study. Stiff, director of Loyola's Cardinal Bernardin Cancer Center, presented findings at the 2013 annual meeting of the American Society of Hematology.

The ASH meeting is the preeminent annual event for physicians and scientists in hematology. Data from more than 5,300 abstracts were presented, and Stiff's abstract was selected as one of the 2013 meeting's top submissions.

Stem cell transplants can save lives of patients who have no other options. Patients receive high-dose chemotherapy, and in some cases, high-dose radiation as well. The treatment, unfortunately, kills healthy blood cells along with the cancerous cells. To rebuild the stores of healthy cells, the patient subsequently receives a transplant infusion of immature stem cells. Over time, these stem cells develop into new blood cells.

Stem cells are produced in the bone marrow. In many cases, patients receive bone marrow stem cells donated by family members or Good Samaritans who have signed up with a bone marrow registry.

But fewer than 50 percent of eligible patients can find a matching bone marrow donor. In such cases, stem cells derived from umbilical cord blood can be an effective alternative because these cells do not require perfect matches. (The cord blood is donated by parents of newborns, and frozen in a cord blood bank.)

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Breakthrough in treating leukemia, lymphoma with umbilical cord blood stem cells

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PUBLIC RELEASE DATE:

11-Dec-2013

Contact: Katya Nasim katya.nasim@kcl.ac.uk 44-207-848-3840 King's College London

Scientists at King's College London have, for the first time, identified the unique properties of two different types of cells, known as fibroblasts, in the skin one required for hair growth and the other responsible for repairing skin wounds. The research could pave the way for treatments aimed at repairing injured skin and reducing the impact of ageing on skin function.

Fibroblasts are a type of cell found in the connective tissue of the body's organs, where they produce proteins such as collagen. It is widely believed that all fibroblasts are the same cell type. However, a study on mice by researchers at King's, published today in Nature, indicates that there are at least two distinct types of fibroblasts in the skin: those in the upper layer of connective tissue, which are required for the formation of hair follicles and those in the lower layer, which are responsible for making most of the skin's collagen fibres and for the initial wave of repair of damaged skin.

The study found that the quantity of these fibroblasts can be increased by signals from the overlying epidermis and that an increase in fibroblasts in the upper layer of the skin results in hair follicles forming during wound healing. This could potentially lead to treatments aimed at reducing scarring.

Professor Fiona Watt, lead author and Director of the Centre for Stem Cells and Regenerative Medicine at King's College London, said: 'Changes to the thickness and compostion of the skin as we age mean that older skin is more prone to injury and takes longer to heal. It is possible that this reflects a loss of upper dermal fibroblasts and therefore it may be possible to restore the skin's elasticity by finding ways to stimulate those cells to grow. Such an approach might also stimulate hair growth and reduce scarring.

'Although an early study, our research sheds further light on the complex architecture of the skin and the mechanisms triggered in response to skin wounds. The potential to enhance the skin's response to injury and ageing is hugely exciting. However, clinical trials are required to examine the effectiveness of injecting different types of fibroblasts into the skin of humans.'

Dr Paul Colville-Nash, Programme Manager for Regenerative Medicine at the MRC, said: 'These findings are an important step in our understanding of how the skin repairs itself following injury and how that process becomes less efficient as we age. The insights gleaned from this work will have wide-reaching implications in the area of tissue regeneration and have the potential to transform the lives patients who have suffered major burns and trauma.'

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Skin's own cells offer hope for new ways to repair wounds and reduce impact of aging on the skin

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A groundbreaking attempt to heal eight Parkinson's patients with their own cells could move from research to the clinic next year.

For eight Parkinson's patients seeking treatment with a new form of stem cell therapy, 2014 promises to be a milestone. If all goes well, next year the FDA will give approval to begin clinical trials. And if the patients can raise enough money, the scientists and doctors working with them will have the money to proceed.

Jeanne Loring, a stem cell scientist at The Scripps Research Institute, discusses the status of a project to treat Parkinson's patients with their own cells, turned into the kind of brain cells destroyed in Parkinson's. The project is a collaboration with Scripps Health and the Parkinson's Association of San Diego.

Scientists at The Scripps Research Institute led by Jeanne Loring have taken skin cells from all patients and grown them into artificial embryonic stem cells, called induced pluripotent stem cells. They then converted the cells into dopamine-making neurons, the kind destroyed in Parkinson's disease.

Loring discussed the project's progress on Friday morning at the 2013 World Stem Cell Summit in San Diego.

If animal studies now under way and other requirements are met, doctors at Scripps Health will perform a clinical trial. They will grow neurons until they are just short of maturity, then transplant them into the brains of the respective patients. The cells are expected to complete maturation in the brain, forming appropriate connections with their new neighbors, and begin making dopamine.

Earlier attempts to treat Parkinson's with a stem cell-like therapy mostly failed because of difficulties in quality control of the source, neural cells from aborted fetuses, Loring said. But some patients gained lasting improvement, a tantalizing hint that the trials were on the right track.

In January, a "pre-pre-IND meeting" is planned with the FDA, Loring said.

Also speaking were Ed Fitzpatrick, one of the eight patients, and Kyoto University researcher Jun Takahashi, who is independently trying the same approach in Japan.

Ed Fitzpatrick, one of eight Parkinson's patients in a program to be treated with his own cells, grown into the kind of brain cells destroyed in Parkinson's.

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Stem cells for Parkinson's getting ready for clinic

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FREEPORT, The Bahamas (PRWEB) December 06, 2013

December 6, 2013 Matt Feshbach, CEO of Okyanos Heart Institute whose mission it is to bring a new standard of care and better quality of life to patients with coronary artery disease using cardiac stem cell therapy, acknowledges the Japanese legislature for its recent approval of a bill aimed at the treatment of certain chronic diseases using regenerative medicine strategies.

The legislation was passed in Japan on November 20th, 2013. The new regenerative medicine law emphasizes the importance of establishing patient safety in the use of adult stem cell therapies prior to being offered commercially. It also serves to support innovation in stem cell and regenerative medicine therapies by providing a framework by which such technologies may be granted new, limited approval paths for some biologics.

Japan has taken a leadership position globally for its passage of enlightened legislation for stem cell therapy, said Feshbach, who recognizes this development as an important milestone in its potential to benefit patients and the field of healthcare.

We applaud Japan as well as other countries including but not limited to Australia, Singapore, and New Zealand for approving stem cell processing devices and/or biologics (such as stem cells) for use in clinics today, he added. This legislation in Japan says that if a stem cell therapy protocol can demonstrate a strong safety profile, physicians have the option to offer it to patients, generally when other standard-of-care interventions have not proven effective and the patients have no other options available to them. Patients will have the choice to use their own stem cells to treat the condition. By tracking the progress of the patients over time, efficacy can be determined and the treatment may become another standard-of-care treatment option available to patients.

While this research is important over the long term, adult stem cell therapy is unique in that it takes advantage of the natural mechanisms of a persons own stem cells to repair the cells, tissues or organs damaged by disease or injury, stated Feshbach. The dawn of a new phase in the evolution of medicine has begun.

Additional countries such as The Bahamas, Panama, Argentina and Jordan have established regulations and legislation designed to both protect patient safety and give access to treatments which have the potential to help unmet needs such as heart failure and other diseases.

Japan represents the second-largest medical market in the world and remains a global leader in both adult stem cell and gene therapy trials. Dr. Shinya Yamanaka, professor and director for the Center for iPS Cell Research and Application (CiRA) at Kyoto University, was awarded a Nobel Prize in 2012 for the discovery of induced pluripotent stem cells (iPS). Click here to read more about the Japanese legislatures recent stem cell measures.

About Okyanos Heart Institute: (Oh key AH nos) Based in Freeport, The Bahamas, Okyanos Heart Institutes mission is to bring a new standard of care and a better quality of life to patients with coronary artery disease using cardiac stem cell therapy. Okyanos adheres to U.S. surgical center standards and is led by Chief Medical Officer Howard T. Walpole Jr., M.D., M.B.A., F.A.C.C., F.S.C.A.I. Okyanos Treatment utilizes a unique blend of stem and regenerative cells derived from ones own adipose (fat) tissue. The cells, when placed into the heart via a minimally-invasive catheterization, stimulate the growth of new blood vessels, a process known as angiogenesis. The treatment facilitates blood flow in the heart and supports intake and use of oxygen (as demonstrated in rigorous clinical trials such as the PRECISE trial). The literary name Okyanos (Oceanos) symbolizes flow. For more information, go to http://www.okyanos.com

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Okyanos Heart Institute CEO Matt Feshbach Congratulates Japan’s Legislators On Stem Cell Bill And Global Regulatory ...

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Dec. 8, 2013 at 2:49 PM ET

Jeff Swensen / for NBC News

The Mogul family at The Children's Institute in Pittsburgh, Pennsylvania where parents Stephen and Robyn have taken their daughter, Bari, 9 and Hayley, 15, to undergoing extensive therapy to help with their rare genetic disorders.

At 15, Hayley Mogul lacks the fine motor skills needed to write. Her sister Bari is 9 and still eating baby food.

There's no cure for their rare disorders, caused by unique genetic mutations. But for once, there's an advantage to having conditions so rare that drug companies cannot even think of looking for a cure. The sisters are taking part in a whole new kind of experiment in which scientists are literally turning back the clock on their cells.

Theyre using an experimental technique to transform the cells into embryonic form, and then growing these baby cells in lab dishes.

The goal is the get the cells to misfire in the lab in just the same way they are in Hayleys and Baris bodies. Its a new marriage of genetics and stem cell research, and represents one of the most promising applications of so-called pluripotent stem cells.

One day these two girls will probably change the face of medicine as we know it, said their father, Steven Mogul.

Steven and Robyn Mogul dont understand why both their daughters ended up with the rare mutations, which cause a range of neurological and metabolic problems.

We have been tested, said Mogul, a 45-year-old wealth manager living in Chicago. We dont have any mutations, and there are no developmental issues. We have no idea how it happened.

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Stem cell science: Can two girls help change the face of medicine?

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NBC News - At 15, Hayley Mogul lacks the fine motor skills needed to write. Her sister Bari is 9 and still eating baby food.

There's no cure for their rare disorders, caused by unique genetic mutations. But for once, there's an advantage to having conditions so rare that drug companies cannot even think of looking for a cure. The sisters are taking part in a whole new kind of experiment in which scientists are literally turning back the clock on their cells.

They're using an experimental technique to transform the cells into embryonic form, and then growing these baby cells in lab dishes.

The goal is the get the cells to misfire in the lab in just the same way they are in Hayley's and Bari's bodies. It's a new marriage of genetics and stem cell research, and represents one of the most promising applications of so-called pluripotent stem cells.

"One day these two girls will probably change the face of medicine as we know it," said their father, Steven Mogul.

Steven and Robyn Mogul don't understand why both their daughters ended up with the rare mutations, which cause a range of neurological and metabolic problems.

"We have been tested," said Mogul, a 45-year-old wealth manager living in Chicago. "We don't have any mutations, and there are no developmental issues. We have no idea how it happened. "

The girls need special schooling and physical therapy. They must wear diapers, and when they get a cold or the flu, they can develop dangerously low blood sugar. "When the kids get sick, get colds or flu, we have to get them to the hospital," Mogul said.

Hayley, 15, has a mutation in a gene called RAI1, which can cause Smith-Magenis syndrome. The syndrome affects 1 in 25,000 people and can disturb sleep patterns, cause obesity and behavioral issues. But Hayley's mutation is unique and puzzling. Bari, 9, has an RAI1 mutation and a similarly unique mutation in the GRIN2B gene, which can cause learning disabilities.

"Bari doesn't talk," Mogul said. "She walks around, she gets around and lets you know what she wants. She is eating baby food and she is drinking from bottles."

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'Something positive for humankind': Girls lend cells to genetic study

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T Cell Therapy for Acute Lymphoblastic Leukemia: Results as of November 2013
CTL019 is a clinical trial of T cell therapy for patients with B cell cancers such as acute lymphoblastic leukemia (ALL), B cell non-Hodgkin lymphoma (NHL), ...

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T Cell Therapy for Acute Lymphoblastic Leukemia: Results as of November 2013 - Video

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Californias government-run stem-cell research agency, on course to spend $3 billion in taxpayer money to find treatments for some of the worlds most intractable diseases, is pushing to accelerate human testing before its financing runs out.

For the California Institute for Regenerative Medicine, time is growing short to fund research that demonstrates the potential of stem cells to help treat everything from cancer to heart disease to spinal cord injuries.

The agency, created by voters in 2004, has given out more than half of its $3 billion from state bonds and must spend the rest by 2017. The largest U.S. funding source for stem-cell research outside the federal government, its under pressure to show results to attract new money from pharmaceutical companies, venture capitalists or even more municipal bonds.

We need to figure out how to keep them going, said Jonathan Thomas, a founding partner of Saybrook Capital LLC in Los Angeles, and chairman of the institutes board, which meets today. We could do public-private partnerships, venture philanthropy, a ballot box.

Embryonic stem cells have the potential to change into any type of cell in the body. They are among the first cells created in embryos after conception. Scientists hope they may replace damaged or missing tissue in the brain, heart and immune system.

California voters approved the bonds after President George W. Bush banned the use of federal funds for research on embryonic stem cells. Since then, other types of stem cells have been shown to act like embryonic cells, relieving some of the debate over the ethics of destroying human embryos to use the cells.

The agencys funding decisions have included a grant of $20 million to a team led by Irv Weissman at the Stanford University School of Medicine, seeking a cure for cancer.

Weissmans team is working on an antibody manufactured with stem cells that allows a cancer patients own immune system to destroy a tumor, instead of relying on toxic radiation or chemotherapy. The antibody counteracts a protein called CD47, which creates what scientists call a dont eat me shield around the cancer. Once that cloak is removed, the patients immune system recognizes the cancer and attacks the tumor, shrinking or eliminating it.

Tests on humans are to begin early next year. The antibody has already worked in mice against breast, colon, ovarian, prostate, brain, bladder and liver cancer.

Two other research projects funded by the California agency are in human trials now -- one targeting HIV, the virus that causes AIDS, and another that regrows cardiac tissue in heart-attack victims.

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Dec. 11, 2013 Muscle cell therapy to treat some degenerative diseases, including Muscular Dystrophy, could be a more realistic clinical possibility, now that scientists have found a way to isolate muscle cells from embryonic tissue.

PhD Student Bianca Borchin and Associate Professor Tiziano Barberi from the Australian Regenerative Medicine Institute (ARMI) at Monash University have developed a method to generate skeletal muscle cells, paving the way for future applications in regenerative medicine.

Scientists, for the first time, have found a way to isolate muscle precursor cells from pluripotent stem cells using a purification technique that allows them to differentiate further into muscle cells, providing a platform to test new drugs on human tissue in the lab. Pluripotent stem cells have the ability to become any cell in the human body including, skin, blood, brain matter and skeletal muscles that control movement.

Once the stem cells have begun to differentiate, the challenge for researchers is to control the process and produce only the desired, specific cells. By successfully controlling this process, scientists could provide a variety of specialised cells for replacement in the treatment of a variety of degenerative diseases such as Muscular Dystrophy and Parkinson's disease.

"There is an urgent need to find a source of muscle cells that could be used to replace the defective muscle fibers in degenerative disease. Pluripotent stem cells could be the source of these muscle cells," Professor Barberi said.

"Beyond obtaining muscle from pluripotent stem cells, we also found a way to isolate the muscle precursor cells we generated, which is a prerequisite for their use in regenerative medicine.

"The production of a large number of pure muscle precursor cells does not only have potential therapeutic applications, but also provides a platform for large scale screening of new drugs against muscle disease."

Using a technology known as fluorescence activated cell sorting (FACS), the researchers identified the precise combination of protein markers expressed in muscle precursor cells that enabled them to isolate those cells from the rest of the cultures.

Ms Borchin said there were existing clinical trials based on the use of specialised cells derived from pluripotent stem cells in the treatment of some degenerative diseases but deriving muscle cells from pluripotent stem cells proved to be challenging.

"These results are extremely promising because they mark a significant step towards the use of pluripotent stem cells for muscle repair," Ms Borchin said.

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Newswise A team led by researchers at Washington University School of Medicine in St. Louis has identified variations in a gene that doubles a persons risk of developing Alzheimers disease later in life.

The research is published online Dec. 11 in the journal Nature.

Over the past two decades, scientists have discovered a number of common genetic variants linked to early-onset (which strikes before age 65) and the more common late-onset forms of Alzheimers disease. But those variants account for only a fraction of Alzheimers cases.

The newly identified variations, found in a gene never before linked to Alzheimers, occur rarely in the population, making them hard for researchers to identify. But theyre important because individuals who carry these variants are at substantially increased risk of the disease.

As part of the new research, the investigators focused on families with several members who had Alzheimers.

As a practical matter, finding mutations linked to Alzheimers disease means it may be possible to identify more people at risk years before they develop any symptoms. These patients could be monitored carefully for early signs of Alzheimers and possibly even get treatments to slow the progression of the disease.

We were very excited to be able to identify a gene that contains some of these rare variants, said lead author Carlos Cruchaga, PhD. And we were surprised to find that the effect of the gene was so large. After adjusting for other factors that can influence risk for the disease, we found that people with certain gene variants were twice as likely as those who didnt have the variants to develop Alzheimers.

As in many genetic studies of Alzheimers, Cruchaga and his co-investigators analyzed DNA from people in families in which multiple members were affected by the disease.

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PUBLIC RELEASE DATE:

11-Dec-2013

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, December 11, 2013People with the genetic blood clotting disorder hemophilia who have been infected with HIV for decades have an increased proportion of immune cells in their blood that specifically target HIV. This protective immune response helps chronically infected hemophilia patients survive, even during periods of HIV activity, according to a study published in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

Volker Daniel and colleagues, University of Heidelberg and Kurpfalz Hospital, Germany, compared the levels of a class of HIV-reactive immune cells called CD8+ lymphocytes in the blood of hemophilia patients infected with HIV for 30 years and in health individuals. They present the results in "HIV-Specific CD8+ T Lymphocytes in Blood of Long-Term HIV-Infected Hemophilia Patients."

"Understanding the reasons for long-term clinical stability in hemophilia patients living with HIV remains an important research goal, with high clinical significance," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "Using a unique cohort of patients, who have been living with HIV-1 for more than 30 years, the authors propose that it is the cellular anti-HIV-1 response in combination with anti-retroviral therapy that ensures the long-term survival of these patients."

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available on the BioResearch Open Access website.

About the Publisher

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Hemophilia and long-term HIV infection -- is there a protective link?

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PUBLIC RELEASE DATE:

11-Dec-2013

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, December 10, 2013Young women, ages 55 years or below, are more likely to be hospitalized for an acute myocardial infarction (AMI) and to die within the first 30 days than men in the same age group, according to a new study published in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://www.liebertpub.com/jwh.

In fact, although overall AMI hospitalization rates declined for both women and men from 2000-2009 in this Canadian study, the only increase was for younger women (<55 years), in whom the AMI rate rose 1.7% per year. Furthermore, Mona Izadnegahdar and coauthors, University of British Columbia and Providence Health Care Research Institute (Vancouver, BC), reported that the higher 30-day mortality rate for young women compared to young men persisted throughout the study period.

"These findings highlight the need for more aggressive strategies to reduce the incidence of AMI and improve outcomes after AMI in younger women," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Complete tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the Official Journal of the Academy of Women's Health and the Society for Women's Health Research.

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Are younger women more likely to have and die from a heart attack?

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Arlington, VA (PRWEB) December 10, 2013

Join us for this free 60-minute webinar on December 12, 2013 at 2:00PM EST/11:00AM PST, as Genetic Engineering & Biotechnology News hosts BioInformatics LLC and other top industry leaders from Life Technologies and Chempetitive Group to discuss effective mobile marketing practices for reaching life scientists.

According to a recent study, one-third of scientists engage in social media either weekly or daily to support their research. And some scientists are constantly using social media for non-work related activities, which indicates that they are also more likely to spend time on social media sites to support their research.

GENs upcoming webinar will tell you what you need to know to reach life scientists who say they want to learn about products and services on mobile devices. Learn specifics about mobile adoption by life scientists, content and messaging unique to the mobile channel, and examples of successful mobile marketing campaigns by a top industry player.

Moderated by: Bill Levine, Director of Digital Media for Genetic Engineering & Biotechnology News

Register for free at the link below: https://cc.readytalk.com/cc/s/registrations/new?cid=h189p3vc56tl

PRESENTER INFO

Bill Kelly, President, BioInformatics LLC Provider of critical market intelligence to major suppliers serving the life science, medical device and pharmaceutical industries.

Jon Young, Senior Manager, Mobile eBusiness, Life Technologies Provider of products and services to leading customers in the fields of scientific research, genetic analysis and applied sciences.

Jeremiah Worth, Director of Digital Strategy, Chempetitive Group Integrated life science marketing agency providing creative, digital, branding and PR services.

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PUBLIC RELEASE DATE:

10-Dec-2013

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, December 10, 2013Antisense therapeutics, a class of drugs comprised of short nucleic acid sequences, can target a dysfunctional gene and silence its activity. A new study has shown that antisense drugs delivered systemically show activity in a wide range of tissues and organs, supporting their broad therapeutic potential in many disease indications, as described in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Nucleic Acid Therapeutics website.

Gene Hung, Xiaokun Xiao, Raechel Peralta, Gourab Bhattacharjee, Sue Murray, Dan Norris, Shuling Guo, and Brett Monia, Isis Pharmaceuticals, Carlsbad, CA, developers of antisense therapeutics, compared two antisense drug chemistries (Generation 2.0 and 2.5) designed to target a gene that is expressed by virtually all cells in mice and non-human primates. They demonstrated antisense activity in many tissues and cell types, including liver, kidney, lung, muscle, adipose, adrenal gland, and peripheral nerves. The Generation 2.5 antisense compound was more effective in a wider range of tissues, according to the results presented in the article "Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals."

"This seminal work addresses one of the most important questions facing the field, the demonstration and evaluation of multiple organ targeting by Nucleic Acid Therapeutics," says Executive Editor Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, MI. "This publication provides a benchmark for convergent analyses in multiple models for preclinical efficacy evaluation."

Nucleic Acid Therapeutics is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC, and C.A. Stein, MD, PhD, City of Hope National Medical Center, Duarte, CA; and Executive Editor Graham C. Parker, PhD.

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About the Journal Nucleic Acid Therapeutics is an authoritative, peer-reviewed journal published bimonthly in print and online that focuses on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. Nucleic Acid Therapeutics is the official journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a free sample issue may be viewed on the Nucleic Acid Therapeutics website.

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New study shows a breadth of antisense drug activity across many different organs

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