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Newswise PHILADELPHIA Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death.

The two main treatments for MPS I are bone marrow transplantation and intravenous enzyme replacement therapy, but these are only marginally effective or clinically impractical, especially when the disease strikes the central nervous system (CNS). Using an animal model, a team from the Perelman School of Medicine at the University of Pennsylvania has proven the efficacy of a more elegant way to restore IDUA levels in the body through direct gene transfer. Their work was published this week online in Molecular Therapy.

The study provides a strong proof-of-principle for the efficacy and practicality of intrathecal delivery of gene therapy for MPS patients, said lead author James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine and director of the Penn Gene Therapy Program. This first demonstration will pave the way for gene therapies to be translated into the clinic for lysosomal storage diseases.

This family of diseases comprises about 50 rare inherited disorders marked by defects in the lysosomes, compartments within cells filled with enzymes to digest large molecules. If one of these enzymes is mutated, molecules that would normally be degraded by the lysosome accumulate within the cell and their fragments are not recycled. Many of the MPS disorders can share symptoms, such as speech and hearing problems, hernias, and heart problems. Patient groups estimate that in the United States 1 in 25,000 births will result in some form of MPS. Life expectancy varies significantly for people with MPS I. Individuals with the most severe form rarely live more than 10 years.

The team used an adeno-associated viral (AAV) vector to introduce normal IDUA to glial and neuronal cells of the brain and spinal cord in a feline model. Their aim was to treat the CNS manifestations of MPS at the source. After a single injection of the AAV9 vector expressing a normal feline IDUA gene sequence and various promoters, the investigators collected blood serum and cerebrospinal fluid (CSF) samples from the test animals and from untreated controls

Some of the treated animals displayed a sharp decline in IDUA levels in the CSF after an initial elevation in the enzyme, which the researchers attribute to an antibody response against IDUA. However, IDUA still persisted at a level sufficient to elicit a positive therapeutic response.

The team also found that one CSF enzyme was elevated in the presence of MPS and propose it could be used as a biomarker for disease activity. All the treated animals displayed a marked decrease in this enzyme, confirming a definite biochemical response to the introduction of the gene vector.

Tissue samples from the brain and spinal cord showed widespread presence of the AAV9 vector throughout all regions of the CNS. IDUA deficiency in the CNS caused by MPS1 results in the accumulation of cholesterol and lipids called gangliosides in brain tissue and accumulation of the sugar glycosaminoglycan in connective tissue and cerebral blood vessels. The animals treated with the AAV9-IDUA vector displayed an almost complete reversal of these molecular markers of MPS.

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Attacking a Rare Disease at its Source With Gene Therapy

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47-Hua -Spinal cord injury (girl, 15 years old) - After stem cell treatment
Hua, a girl, 15 years old, happened to meet a car cash, which caused the movement dysfunction of her lower limbs, sensation disorder, urination and bowl move...

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47-Hua -Spinal cord injury (girl, 15 years old) - After stem cell treatment - Video

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29 1 Mr. Kang -Burst Fracture, Spinal Cord Injury (Male, 35-year-old) Before stem cell treatment

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29 1 Mr. Kang -Burst Fracture, Spinal Cord Injury (Male, 35-year-old) Before stem cell treatment - Video

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28 1 Mrs. Zhou -C6-7 Spinal Cord Injury (Female)
Mrs. Zhou, suffered from C6-7 spinal cord injury 4 years ago. She received 1st round stem cell treatment in March, 2010. Before the 1st round stem cell treat...

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28 1 Mrs. Zhou -C6-7 Spinal Cord Injury (Female) - Video

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Therapeutic Interventions- International Center for Spinal Cord Injury at Kennedy Krieger Institute
The International Center for Spinal Cord Injury (ICSCI) at Kennedy Krieger Institute was founded on the philosophy that individuals with paralysis can always hope for recovery of sensation,...

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Therapeutic Interventions- International Center for Spinal Cord Injury at Kennedy Krieger Institute - Video

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Sheryl Ubelacker, The Canadian Press Published Tuesday, August 26, 2014 6:45AM EDT

TORONTO -- Canadian doctors have begun using stem cell transplants to treat "stiff person syndrome," a rare neurological condition in which a patient's leg and other muscles suddenly contract painfully, often leaving them immobilized like a tin soldier.

The disorder, which affects an estimated one in a million people, occurs when the immune system turns against a person's own tissues, in this case attacking cells in the brain and spinal cord.

Stem cell transplants have been used to treat patients with other auto-immune diseases, among them multiple sclerosis, scleroderma and Crohn's disease, but this may be the first time the procedure has been employed to alleviate the symptoms of stiff person syndrome, or SPS, the researchers reported Monday in the journal JAMA Neurology.

SPS is characterized by episodes of stiffness in the muscles and painful muscle spasms, which can be brought on by stress, loud noises or emotional distress. Some people with the disorder are so disabled they are unable to walk or move and may isolate themselves at home to avoid triggering an attack.

"Sometimes this happens when they're startled," said Dr. Harry Atkins of the Blood and Marrow Transplant Program at the Ottawa Hospital, who headed a team that transplanted stem cells into two women with the disease.

"So you can imagine walking across the street and someone honks the horn and you can't move, or you start falling and because your muscles can't move, you just fall and you hurt yourself," Atkins said Monday from Ottawa.

"It really does provide a barrier with just going on with your life."

Tina Ceroni of Toronto is one of the two SPS patients who had the stem-cell transplant -- and she said it has given back her life.

The personal fitness trainer, now 36, started getting severe symptoms in her late 20s. Initially she was diagnosed with hyponatremia, or low blood sodium, thought to be related to her heavy training schedule for a half-ironman competition.

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Canadian doctors use stem cells to treat 'stiff person syndrome'

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The heart contains four different chambers and different cell types such as cardiomyocytes (CMs), endocardial cells (ECs) covering the inner layer of the heart, epicardial cells covering the outer layer of the heart and smooth muscle cells (SMCs) covering the coronary arteries and main vessels. During embryonic development, the cells that will form the heart need to be specified at the correct time, migrate at the correct place, proliferate to ensure the harmonious morphogenesis and growth of the heart. Any defects during this critical stage of development will lead to congenital heart diseases, which represent the first cause of severe birth malformations. While different progenitors that contribute to the development of the heart have been identified, it remains unclear whether these cells arise from common progenitors or derive from distinct progenitors that are specified at different time during development.

In a new study published in Nature Cell Biology, researchers led by Pr. Cdric Blanpain, MD/PhD, WELBIO investigator at the IRIBHM, Universit libre de Bruxelles, Belgium, have identified temporally distinct populations of cardiac progenitors that differentiate into different cell lineages and contribute to different regions of the heart.

Fabienne Lescroart, Samira Chabab and colleagues performed for the first time a temporally controlled clonal analysis of early cardiac progenitors, in which they marked single cells at the early stages of embryonic development and assess the contribution of single cardiac progenitors to the heart development. In contrast to the prevalent notion that these cells arise from a common progenitors, the researchers found that the different cardiac progenitors are specified at different time points during development and will only contribute to the morphogenesis of certain cardiac regions, like if the heart is build from different blocs that are made at different time during development. Furthermore, the researchers found that in contrast to the multilineage differentiation of these cells in vitro, the early population of cardiac progenitors did not differentiate into all cardiovascular lineages in vivo, but were rather pre-specified to give rise to either cardiac cells or endocardial cells, suggesting that the ultimate fate of the progenitors can be regulated by the environmental cues that the different progenitors encounter during cardiac morphogenesis. "We were extremely surprized to find that the early the cardiac progenitors have a much narrow regional contribution and were not able to differentiate into more than one cell types in contrast to late born cardiac progenitors. We need to completely rethink about the way heart is formed" comment Fabienne Lescroart, the first author of the study.

Using new tools to isolate for the first time the early cardiac progenitors during embryonic development, Fabienne Lescroart, Samira Chabab and colleagues define the molecular characteristics of these different progenitors and showed that the different populations of Mesp1 progenitors, although very similar molecularly, present also notable difference, consistent with their lineage and regional contribution. In addition, characterization of the gene expression at a single cell level have shown that the cardiac progenitors were molecularly heterogenous and expressed different combination of genes that will define the cell fate and regionalization of each progenitors. Understanding how this specificity is achieved will be important to instruct and/or restrict the fate of multipotent cardiovascular progenitors into a particular cell lineage in vivo. The answers to these questions will be important to design new strategies to direct the differentiation of pluripotent cells and iPS cells specifically into pure population of cardiac cells, and for improving cellular therapy in cardiac diseases.

In conclusion, this work uncovers how the heart is build from temporally distinct progenitors with different differentiation potential. This work provides the first temporal clonal analysis of heart development and the first molecular characterization of cardiac progenitors at the early step of cardiac morphogenesis. " This new study really changes the way we think about cardiac development and have important implications for better understanding the aetology of congenital cardiac malformations and should be the starting point of further studies to understand how the regionalization and the choice of differentiation into a particular cardiovascular lineage is achieved, which have important implications for improving cell therapy during cardiac repair" comments Pr Cdric Blanpain, the senior author of this study.

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The above story is based on materials provided by Libre de Bruxelles, Universit. Note: Materials may be edited for content and length.

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Early lineage segregation during early mammalian heart development defined by researchers

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PUBLIC RELEASE DATE:

25-Aug-2014

Contact: Justin Jackson jjackson@burnsmc.com 212-213-0006 x327 Burns McClellan

Santa Monica, CA August 25, 2014 Kite Pharma, Inc., (NASDAQ: KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, today announced the publication of clinical results in a cohort of patients demonstrating the potential to treat aggressive non-Hodgkin's lymphoma with an anti-CD19 chimeric antigen receptor (CAR) T cell therapy. Kite's most advanced product candidate, KTE-C19, is an anti-CD19 CAR T cell therapy that involves genetically modifying a patient's T cells to express a CAR that is designed to target CD19, a protein expressed on the cell surface of B cell lymphomas and leukemias.

The findings from an ongoing Phase 1-2a clinical trial funded by Kite and conducted by the Surgery Branch of the National Cancer Institute (NCI) demonstrated that in 12 out of 13 evaluable patients with advanced B-cell malignancies, administration of anti-CD19 CAR T cells resulted in complete remission in eight patients and partial remission in four patients, representing an overall objective response rate of 92%. Of seven evaluable patients with chemotherapy-refractory DLBCL, four achieved complete remission, three of which are ongoing with durations ranging from 9 to 22 months. These findings are being published in an article titled, "Chemotherapy-refractory Diffuse Large B-cell Lymphoma and Indolent B-cell Malignancies Can Be Effectively Treated with Autologous T Cells Expressing an Anti-CD19 Chimeric Antigen Receptor," DOI: 10.1200/JCO.2014.56.2025, which is appearing in the August 25, 2014 issue of the American Society of Clinical Oncology's Journal of Clinical Oncology.

Kite and the Surgery Branch of the NCI, led by Steven A. Rosenberg, M.D., Ph.D., are collaborating under a Cooperative Research and Development Agreement (CRADA) for the research and development of eACT based product candidates for the treatment of multiple cancer indications. The reported Phase 1-2a clinical trial is being conducted at the NCI with Dr. Rosenberg serving as principal investigator. Additional authors of the published study include James N. Kochenderfer, M.D., who presented earlier data for the NCI from the trial at the 55th American Society of Hematology (ASH) Annual Meeting in December 2013.

David Chang, M.D., Ph.D., Kite Pharma's Executive Vice President, Research and Development, and Chief Medical Officer, commented, "To date, Kite and the NCI have conducted an extensive program to investigate personalized T cell immunotherapies for blood cancers and solid tumors, including in patients with refractory DLBCL. Both the high overall response rate and the durability of the complete remissions are noteworthy, and we believe our anti-CD19-CAR T cell approach holds great potential for the treatment of B cell malignancies, including those with aggressive, resistant disease for which there are no viable treatment options."

Ronald Levy, M.D., Professor of Medicine, Director of the Lymphoma Program and Former Chief of the Division of Oncology at Stanford University and member of Kite's Scientific Advisory Board, commented, "I have been impressed by the results reported and updated from Dr. Rosenberg's group at the NCI. Particularly compelling are the frequency and the duration of the responses obtained in the difficult-to-treat patient population with relapsed, refractory lymphomas." Dr. Levy serves as a consultant to Kite and is helping to guide the Company in their upcoming clinical trials.

"We are greatly encouraged by the strong results we have seen from our joint lead clinical program with the NCI," commented Arie Belldegrun, M.D., FACS, Kite's President and Chief Executive Officer. "Based on this substantial progress, Kite plans to file an IND in the fourth quarter of this year to initiate a Phase 1-2 single-arm multicenter clinical trial of KTE-C19 in patients with DLBCL who have failed two or more lines of therapy. We are excited to advance this promising therapy and anticipate commencing patient enrollment in our DLBCL clinical trial in the first half of 2015."

Key Study Findings

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Kite Pharma announces positive results in patients with aggressive non-Hodgkin's lymphoma

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Toronto, ON (PRWEB) August 26, 2014

Bel Marra Health, who offers high-quality, specially formulated vitamins and nutritional supplements, has reported on new research that has shown the success of the first ever biological pacemaker that could put an end to invasive surgeries.

As Bel Marra Health reports in its article, (http://www.belmarrahealth.com/heart-health/heart-patients-to-live-longer-thanks-to-new-gene-therapy/), the study was conducted by Los Angeles Cedars-Sinai Heart Institute and published in Science Translational Medicine in July. For this study, 12 pigs with heart block a condition where the electrical signal is slowed or disrupted as it moves through the heart were injected with either the single gene, called TBX18, to reprogram cells, or a fluorescent green protein acting as a placebo.

The patch of peppercorn-sized cells acted as a pacemaker for a two-week period, performing the function of a conventional one. During this same period, cardiologists looked at the average heart rate of the pigs in the morning when they ate and at night when they slept.

They found that the gene therapy was fast-acting, reprogramming enough muscle cells to effectively regulate heart rate within 24 to 48 hours. After eight days of testing, the average heart rate was much higher in the pigs that received the therapy than ones that did not.

This biological pacemaker, as its been dubbed by researchers, could be useful for certain patients, such as those who develop infections from electronic pacemakers and need to have the devices temporarily removed, or fetuses with life-threatening heart disorders who cannot have an electronic pacemaker implanted.

Spokesperson for Bel Marra Health, Dr. Victor Marchione, says, Since the early 1960s, pacemakers have been widely available, and theyve constantly improved, becoming more safe and sophisticated.

Conventional pacemakers are electronic, implanted into the chest to control an abnormal heartbeat. Electronic pacemakers restore regular function to slowing and arrhythmic hearts by using electricity to stimulate heartbeats. Thats a function usually performed by a cluster of thousands of cardiac cells that tell the heart to pump at a regular rate.

These mechanisms are lifesaving for many people with abnormal or slow heart rhythms. But they require an invasive surgery to be installed. So scientists have been waiting for the day when an implant is no longer needed by patients.

Of course, the applications of this new research are still a long way off. And the benefits of a pacemaker usually outweigh the risks. Still, pig hearts are similar to human hearts in their size and the way they work, so theres good reason to think that the new findings could translate to humans.

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Bel Marra Health Reports on the Success of New Gene Therapy for Heart Patients

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Dresden, Germany (PRWEB) August 26, 2014

Cenix BioScience GmbH, a leading preclinical contract research provider and technology developer specialized in genome-scale gene modulation studies and high throughput, high content pharmacology, today announced that it has signed a research framework agreement with Metanomics Health GmbH, a BASF Group company, focused on comprehensive metabolite profiling services and the development of metabolomic biomarkers.

As the agreements first work assignment, Metanomics Health has commissioned Cenix to undertake a target validation project for an undisclosed disease indication. Under the jointly developed work plan, Cenix will leverage its longstanding industry-leading expertise in high content screening, utilizing the Definiens XD image analysis platform, for comprehensive functional characterization of target genes designated by Metanomics Health.

We foresee very strong synergies between our own capabilities and the exciting approach taken by Metanomics Health towards biomarker discovery and validation, said Dr. Christophe Echeverri, CEO/CSO of Cenix. We appreciate and value the trust of our colleagues at Metanomics Health and look forward to advancing their programs."

Financial terms were not disclosed.

About Cenix BioScience With operations in Germany and the U.S., Cenix BioScience conducts contract research and develops new reagent technologies focused on a wide array of preclinical cell-based and in vivo applications including RNAi-based gene silencing, miRNA modulation, compound testing, advanced genomics analyses and high content screening to accelerate drug discovery and development. Now in its 15th year, Cenix has established unrivaled scientific and commercial track records in this field, successfully advancing therapeutic programs for numerous industry and academic partners in a broad range of disease fields. This success, illustrated by a high rate of repeat business from top clients and an ever-growing list of new clients, is driven by the consistent application of industrial best practices through highly customized, multi-staged projects. Their careful design yield maximal strategic value and accountability, with minimized risk and full data transparency. More information: http://www.cenix.com.

About Metanomics Health Metanomics Health GmbH, a BASF Group company, was founded in 2003 and is a leader in robust, non-targeted and targeted comprehensive metabolite profiling services (metabolomics) to healthcare customers. Serving pharma, nutrition and diagnostics companies, as well as academic partners, Metanomics Health state-of-the-art biomedical data interpretation, combined with innovative bioinformatics and data mining systems, enables discovery and validation of both simple and complex metabolomic biomarkers. Metanomics Health delivers novel biomarkers of disease and drug efficacy, increased mechanistic understanding of drug action and adverse effects, as well as insights into underlying pathways to its partners, thereby providing actionable results for healthcare research and development programs. More information: http://www.metanomics-health.de.

Contact Dr. Christoph Sachse Director Cell-based Services Cenix BioScience GmbH E: info(at)cenix(dot)com W: http://www.cenix.com

Cenix and the Cenix BioScience logo are registered trademarks owned by Cenix BioScience GmbH. Definiens XD and Definiens are registered trademarks owned by Definiens AG.

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Cenix BioScience and Metanomics Health Sign Research Agreement

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In a recent report in Aging Cell, a multidisciplinary team of Spanish scientists, led by Tim Cash and Manuel Serrano at the Spanish National Cancer Research Centre (CNIO), identify rare variants in the APOB gene in several families where exceptional longevity (more than 100 years of age) appears to cluster.

Investigators identified three Spanish families with at least two siblings of around 100 years of age and they sequenced their genes in the hope of finding rare variants that could be associated with extreme longevity.

Remarkably, only one gene was found carrying rare variants in all the long-lived siblings of the three families, namely, APOB.

APOB is an attractive longevity gene because of its previous link to hypobetalipoproteinemia, a putative "longevity syndrome" and also because the protein encoded by APOB works in lipid transport together with the related protein APOE, which has common genetic variants with undisputed assocations with longevity.

This work is a first step in the identification of the genetic basis of familial extreme longevity and it points to cholesterol and lipid metabolism as an important determinant of human longevity.

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The above story is based on materials provided by Centro Nacional de Investigaciones Oncologicas (CNIO). Note: Materials may be edited for content and length.

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APOB, a gene involved in lipid transport, linked to cases of familial extreme longevity

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PUBLIC RELEASE DATE:

25-Aug-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, August 25, 2014In a closed-loop control approach to managing type 1 diabetes, glucose sensors placed under the skin continuously monitor blood sugar levels, triggering the release of insulin from an implantable insulin pump as needed. The aim of this closed-loop insulin delivery system is improved control of blood glucose levels throughout the day and night. But a new study in adults and adolescents found that mean blood glucose levels remained at safe levels 53-82% of the time, according to the results published in Diabetes Technology & Therapeutics (DTT), a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the DTT website at http://online.liebertpub.com/doi/full/10.1089/dia.2014.0066 until September 25, 2014.

Howard Zisser, MD and an international team of researchers representing the Control to Range Study Group measured plasma glucose levels every 15-30 minutes in a group of individuals with type 1 diabetes who participated in the "Control to Range" multinational artificial pancreas study. They monitored the adults and teens over 22 hours, including three meals and periods of day and night. The authors describe the risks of hypo- and hyperglycemia, the variability between participants, and the differences in daytime/nighttime results, and also propose improvements needed in the design and implementation of closed-loop systems in the article "Multicenter Closed-Loop Insulin Delivery Study Points to Challenges for Keeping Blood Glucose in a Safe Range by a Control Algorithm in Adults and Adolescents with Type 1 Diabetes from Various Sites".

"It appears that we are getting closer to an Artificial Pancreas option for patients with type 1 diabetes," says DTT Editor-in-Chief Satish Garg, MD, Professor of Medicine and Pediatrics at the University of Colorado Denver. "The first version may need to be a hybrid system in which meals and exercise are announced with necessary dose adjustments along with Automatic Threshold Suspend for hypoglycemia."

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About the Journal

Diabetes Technology & Therapeutics (DTT) is a monthly peer-reviewed journal that covers new technology and new products for the treatment, monitoring, diagnosis, and prevention of diabetes and its complications. Led by Editor-in-Chief Satish Garg, MD, Professor of Medicine and Pediatrics at the University of Colorado Denver, the Journal covers topics that include noninvasive glucose monitoring, implantable continuous glucose sensors, novel routes of insulin administration, genetic engineering, the artificial pancreas, measures of long-term control, computer applications for case management, telemedicine, the Internet, and new medications. Tables of content and a sample issue may be viewed on the Diabetes Technology & Therapeutics (DTT) website at http://www.liebertpub.com/DTT. DTT is the official journal of the Advanced Technologies & Treatments for Diabetes (ATTD) Conference.

About ATTD

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Do closed-loop insulin delivery systems improve blood glucose control in type 1 diabetes?

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PUBLIC RELEASE DATE:

25-Aug-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, August 25, 2014 Nearly 1 in 6 adults worldwide may suffer from chronic constipation and, over time, the disorder can cause serious complications. Auriculotherapy, a form of acupuncture that involves stimulating targeted points on the outer ear, may help in managing constipation. Evidence from numerous clinical studies published between 2007-2013 that evaluated the effectiveness of auriculotherapy in treating patients with constipation is presented and discussed in a Review article in The Journal of Alternative and Complementary Medicine, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on The Journal of Alternative and Complementary Medicine website until September 25, 2014.

Li-Hua Yang and coauthors from the Hospital of Nanjing University of Traditional Chinese Medicine, Jiangsu Province Hospital of Traditional Chinese Medicine, and Southeast University School of Public Health, Nanjing, China, analyzed the results of 17 published studies, comparing the effectiveness of auriculotherapy in managing and relieving constipation and in alleviating symptoms associated with constipation between affected patients and a control group. The authors present their data and conclusions in the article "Efficacy of Auriculotherapy for Constipation in Adults: A Systematic Review and Meta-Analysis of Randomized Controlled Trials".

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About the Journal

The Journal of Alternative and Complementary Medicine is a monthly peer-reviewed journal publishing observational, clinical, and scientific reports and commentary intended to help healthcare professionals and scientists evaluate and integrate therapies into patient care protocols and research strategies. Complete tables of content and a sample issue may be viewed on The Journal of Alternative and Complementary Medicine website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Alternative and Complementary Therapies, Medical Acupuncture, Brain and Gut, and Journal of Medicinal Food. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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Can auriculotherapy help relieve chronic constipation?

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By Tom Abate

Stanford bioengineer Christina Smolke has been on a decade-long quest to genetically alter yeast to "brew" opioid medicines in stainless steel vats, eliminating the need to raise poppies.

For centuries poppy plants have been grown to provide opium, the compound from which morphine and other important medicines such as oxycodone are derived.

Now bioengineers at Stanford have hacked the DNA of yeast and reprogrammed these simple cells to make opioid-based medicines via a sophisticated extension of the basic brewing process that makes beer.

Led by bioengineering Associate Professor Christina Smolke, the Stanford team has already spent a decade genetically engineering yeast cells to reproduce the biochemistry of poppies, with the ultimate goal of producing opium-based medicines, from start to finish, in fermentation vats.

"We are now very close to replicating the entire opioid production process in a way that eliminates the need to grow poppies, allowing us to reliably manufacture essential medicines while mitigating the potential for diversion to illegal use," said Smolke, who outlines her work in the Aug. 24 edition of Nature Chemical Biology.

In the new report, Smolke and her collaborators, Kate Thodey, a postdoctoral scholar in bioengineering, and Stephanie Galanie, a doctoral student in chemistry, detail how they added five genes from two different organisms to yeast cells. Three of these genes came from the poppy itself, and the others from a bacterium that lives on poppy plant stalks.

This multi-species gene mashup was required to turn yeast into cellular factories that replicate two, now separate processes: how nature produces opium in poppies, and then how pharmacologists use chemical processes to further refine opium derivatives into modern opioid drugs such as hydrocodone.

Morphine is one of three principal painkillers derived from opium. As a class they are called opiates. The other two important opiates are codeine, which has been used as a cough remedy, and thebaine, which is further refined by chemical processes to create higher-value therapeutics such as oxycodone and hydrocodone, better known by brand names such as OxyContin and Vicodin, respectively.

Today, legal poppy farming is restricted to a few countries including Australia, France, Hungary, India, Spain and Turkey supervised by the International Narcotics Control Board, which seeks to prevent opiates like morphine, for instance, from being refined into illegal heroin.

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Stanford bioengineers close to brewing painkillers without opium from poppies

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Sheryl Ubelacker, The Canadian Press Published Tuesday, August 26, 2014 6:45AM EDT

TORONTO -- Canadian doctors have begun using stem cell transplants to treat "stiff person syndrome," a rare neurological condition in which a patient's leg and other muscles suddenly contract painfully, often leaving them immobilized like a tin soldier.

The disorder, which affects an estimated one in a million people, occurs when the immune system turns against a person's own tissues, in this case attacking cells in the brain and spinal cord.

Stem cell transplants have been used to treat patients with other auto-immune diseases, among them multiple sclerosis, scleroderma and Crohn's disease, but this may be the first time the procedure has been employed to alleviate the symptoms of stiff person syndrome, or SPS, the researchers reported Monday in the journal JAMA Neurology.

SPS is characterized by episodes of stiffness in the muscles and painful muscle spasms, which can be brought on by stress, loud noises or emotional distress. Some people with the disorder are so disabled they are unable to walk or move and may isolate themselves at home to avoid triggering an attack.

"Sometimes this happens when they're startled," said Dr. Harry Atkins of the Blood and Marrow Transplant Program at the Ottawa Hospital, who headed a team that transplanted stem cells into two women with the disease.

"So you can imagine walking across the street and someone honks the horn and you can't move, or you start falling and because your muscles can't move, you just fall and you hurt yourself," Atkins said Monday from Ottawa.

"It really does provide a barrier with just going on with your life."

Tina Ceroni of Toronto is one of the two SPS patients who had the stem-cell transplant -- and she said it has given back her life.

The personal fitness trainer, now 36, started getting severe symptoms in her late 20s. Initially she was diagnosed with hyponatremia, or low blood sodium, thought to be related to her heavy training schedule for a half-ironman competition.

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Canadian doctors use stem cells to treat 'stiff person syndrome'

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ABC Kellie van Meurs (3rd from R) died of a heart attack last month while receiving stem cell treatment in Moscow.

Rogue operators in Australia and overseas are charging thousands of dollars for ineffectual stem cell treatments, a leading stem cell research group has warned.

And Stem Cells Australia says there is a growing number of patients going overseas for stem cell treatments which are limited in Australia.

A loophole in the therapeutic goods legislation means that doctors are legally allowed to treat patients, both here and overseas, with their own stem cells even if that treatment is unsafe or has not been proven effective through clinical trials.

Stem Cells Australia believes that dozens of doctors in Australia offer the questionable treatments.

"They're selling treatment without any proof of benefit, and without any proof of safety," Associate Professor Megan Munsie, a stem cell biologist at the University of Melbourne, told 7.30.

Annie Leverington was diagnosed with multiple sclerosis in 2007.

She was once a talented flamenco dancer and worked as a court stenographer.

But in 2002 she noticed something was wrong when her fingers started to "drop" during long trials.

Then her feet started to go.

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Rogue stem cell therapy operators charging thousands for ineffective treatments, researchers say

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Update On Stem Cell Research
Regenerative medicine and stem cell technology is revolutionizing the medical field. Kristin Comella, Chief Science Officer for Bioheart (BHRT), explains the growth of the industry and the potential.

By: Bioheart Inc.

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Update On Stem Cell Research - Video

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Learn about Bioheart Inc. (BHRT)
Kristin Comella, Chief Science Officer for Bioheart (BHRT), discusses the company #39;s research using stem cell technology and regenerative medicine products.

By: Bioheart Inc.

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Learn about Bioheart Inc. (BHRT) - Video

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Prof Noel Caplice, director of the Centre for Research in Vascular Biology at University College Cork, displays his stent mesh. Photograph: Michael MacSweeney/Provision

As Prof Noel Caplice describes it, a revolutionary new system that avoids putting patients through heart bypass operations was literally a back-of- the-garage effort.

A cardiologist in Cork, he came up with the treatment when working as a cardiologist at the Mayo Clinic seven years ago. During this time, Caplice and an engineer friend worked on prototype meshes and attaching these to stents.

The treatment introduces cells that encourage the body to make new blood vessels that grow past the blockage, actually reversing the disease in as little as three or four weeks.

The treatment may also offer hope for patients suffering from other cardiovascular disorders such as peripheral artery disease, a common risk in diabetes. And, because it uses the patients own cells, there is no question of rejection, says Caplice, director of University College Corks Centre for Research in Vascular Biology.

This would represent a major step forward in the treatment of coronary artery disease, he adds. Instead of open-heart surgery and stitching in arteries to bypass a blockage, it causes the body to grow its own bypass. He is leading the research, which also involves the Mayo Clinic in the US, and the team has published a paper describing the work in the current issue of the journal Biomaterials.

He came up with the idea when working as a cardiologist at the Mayo Clinic seven years ago, he says.

One area we were interested in was patients who were inoperable, patients who were too ill to face open-heart surgery and who had no options. That represents about 20 to 25 per cent of all patients with coronary artery disease.

He was a scientist physician while at the Mayo as he is now, doing research but also working with patients, and he ran his own laboratory. He originally thought of introducing stem cells to encourage blood vessel growth, but when injected they go everywhere, you cant direct them in the body.

Caplice is also a consultant cardiologist at Cork University Hospital.

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Bypassing surgery for new cardiac treatment

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Q: My daughter plays college soccer and ruptured her ACL. The coaches mentioned stem cell injections that some big-name athletes have used to recover from injuries. Should we consider them? Paige R., Chicago

A: Professional athletes are always looking for the fastest way to heal their injuries. In 2010, Yankees pitcher Bartolo Colon was treated for a torn rotator cuff with injections of fat and bone marrow adult stem cells; he's playing for the Mets this season. And in 2011, Denver Broncos' Peyton Manning opted for injections of his own fat stem cells to try to get over a neck injury. Two years later, he had a record-breaking season and took the Broncos to the Super Bowl. But does this mean the injections worked? Nope.

Colon's agent attributes the pitcher's career turnaround to a re-dedication to the game, not the injections, and Manning followed up his stem cell treatment with major surgery and intense rehab (done in secret).

There's just no solid evidence yet that injections of adult bone marrow (or fat) stem cells effectively regenerate and repair damaged tendons or ligaments, and you cannot be certain of what the injections contain or their side effects. They often are delivered in an unregulated environment and aren't FDA-approved.

We suspect your daughter is headed for reconstructive surgery and six months of rehab. Then she needs to learn new ways to move so she reduces stress on her knees. One metastudy found that two ACL-injury-prevention regimens were effective: Sportsmetrics promotes leg and core strength, increases vertical jump height and may improve speed and agility; the Prevent Injury and Enhance Performance (PEP) program, makes a big difference in the flexion strength of the knee. Both improve athletic performance tests and reduce injury rates. Rehab is tough, and there are no shortcuts, but we bet your daughter has the grit to do it!

Q: I had Roux-en-Y gastric bypass surgery last year, and my blood glucose levels were almost normal even before I lost any weight. How is that possible? Marty Z., Jupiter, Florida

A: Congrats, Marty. We hope you're continuing to have such good results. It is amazing that bypassing part of your stomach and intestine could have such an immediate effect on your blood sugar levels, and just recently researchers have figured out why that happens sometimes. It seems to have something to do with the bacteria that live in your digestive tract, also called your gut biome.

Roux-en-Y surgery bypasses most of the stomach and the first part of the small intestine, called the duodenum. That's where a lot of your gut bacteria live and where they influence gut hormones that regulate appetite, insulin use, glucose levels and more. So right away, the surgery decreases levels of hormones that regulate appetite; you can eat less without being hungry. That alone lowers glucose levels and increases the effectiveness of your body's insulin supply.

Also, when you have diabetes, your gut bacteria are thrown out of balance; the bad guys overwhelm the good guys (like bifidobacteria and lactobacillus). And that means the hormones that affect how cells get and use glucose can't do their job. The glucose stays in your bloodstream instead of being used as fuel by your cells. But once a lot of the bad gut bacteria are bypassed, your gut biome snaps back into balance and the bacteria and hormones work together to regulate blood sugar levels.

So we suggest you keep your gut biome balanced and happy with a healthy diet of five or more servings of fruits and veggies a day. Asparagus, garlic, cooked onions and dandelion greens deliver prebiotics that help good-for-you gut bacteria thrive. Fermented foods like nonfat kefir and kimchi contain healthful probiotics. Also, avoid saturated fats and added sugars they just make your biome miserable. And we like daily spore probiotic supplements containing bacillus coagulans GBI-30, 6086 and lactobacillus GG.

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Stem cells for sports injuries; gastric bypass and the gut biome

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British scientists produced a working thymus, a vital immune system "nerve centre" located near the heart.

In future the technique, so far only tested on mice, could be used to provide replacement organs for people with weakened immune systems, scientists believe.

But it might be another 10 years before such a treatment is shown to be effective and safe enough for human patients.

The research by-passed the usual step of generating "blank slate" stem cells from which chosen cell types are derived.

Instead, connective tissue cells from a mouse embryo were converted directly into a completely different cell strain by flipping a genetic "switch" in their DNA.

The resulting thymic epithelial cells (TECs) were mixed with other thymus cell types and transplanted into mice, where they spontaneously organised themselves and grew into a whole structured organ.

Professor Clare Blackburn, from the Medical Research Council (MRC) Centre for Regenerative Medicine at the University of Edinburgh, who led the team of scientists, said: "The ability to grow replacement organs from cells in the lab is one of the 'holy grails' in regenerative medicine. But the size and complexity of lab-grown organs has so far been limited.

"By directly reprogramming cells we've managed to produce an artificial cell type that, when transplanted, can form a fully organised and functional organ. This is an important first step towards the goal of generating a clinically useful artificial thymus in the lab."

If the immune system can be compared with an army, the thymus acts as its operations base. Here, T-cells made in the bone marrow are primed to attack foreign invaders, just as soldiers are armed and briefed before going into battle.

Once deployed by the thymus, the T-cells protect the body by scanning for infectious invaders such as bacteria and viruses, or dangerous malfunctioning cells, for instance from tumours. When an "enemy" is detected, the T-cells mount a co-ordinated immune response that aims to eliminate it.

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First intact organ built from cells created in lab

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Researchers at the University of Texas (UT) Southwestern Medical Center have developed a technique that corrects a mutation leading to Duchenne muscular dystrophy (DMD). The technique, called CRISPR/Cas9-mediated genome editing, removes the mutation entirely in mice, and could have far-reaching consequences in the treatment of muscular dystrophy in people.

According to the Centers for Disease Control, DMD appears in approximately one out of every 3,500 male births in the US (but rarely appears in girls). It usually strikes before the age of six, often confining patients to a wheelchair before adolescence, with death generally before age 25.

It is a severe form of muscular dystrophy caused by a mutation in a gene called dystrophin that leads to loss of function and strength, not only in voluntary muscles such as those in the arms and legs, but also (later) in the cardiovascular system. It has no cure and existing treatments focus on improving quality of life more so than on halting the progression of the disease.

Using CRISPR/Cas9-mediated genome editing to precisely remove the mutation in DNA responsible for DMD, the UT team found that the mouse's DNA repair mechanisms replace it with a normal copy of the gene. Unlike other approaches, such as exon skipping (causing cells to "skip" the mutation) and gene therapy (which delivers functional dystrophin via a harmless virus but retains the original dysfunctional copy of the gene), this new technique could potentially correct the problem at the source. In other words, it could permanently fix genetic defects, thereby promising completely functional DNA protein. This, in turn, could have a big impact on muscle regeneration over time.

"At the moment, we still need to overcome technical challenges, in particular to find better ways to deliver CRISPR/Cas9 to the target tissue and to scale up," says Dr. Eric Olson, director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern. "But in the future we might be able to use this technique therapeutically, for example to directly target and correct the mutation in muscle stem cells and muscle fibers."

Mice injected with even just a subset of corrected cells showed progressive, widespread improvement over time, the research found. The hope is that the technique can one day be adapted for human treatment of muscular dystrophy.

"This is very important for possible clinical application of this approach in the future," Dr. Olson explains. "Skeletal muscle is the largest tissue in the human body and current gene therapy methods are only able to affect a portion of the muscle. If the corrected tissue can replace the diseased muscle, patients may get greater clinical benefit."

The research is described in a paper published in the journal Science.

Source: UT Southwestern

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New gene editing method corrects muscular dystrophy in mice

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Genetic research will be in the spotlight next week when the most advanced genome sequencing facility in the Southern Hemisphere opens its doors to the public.

A free public forum at The University of Queensland Diamantina Institute (UQDI) on Monday, 1 September will feature speakers highlighting medical, legal and ethical implications of advances in genome sequencing.

Advances in genome sequencing allow health practitioners to better understand the genetic causes of many common human diseases.

UQDI Director Professor Matthew Brown said the forum would bean excellent opportunity for members of the public to speak with genomics experts and learn more about genetic research being undertaken in Brisbane.

Were keen to communicate the rapid advances being made in genomic research and the opportunities that genome sequencing presents for tailoring medical treatment to suit individuals, he said.

This is a great opportunity to showcase the work of the UQ Centre for Clinical Genomics and help people understand how research conducted right here in Brisbane will benefit us all.

Professor Brown said Health Minister Mr Lawrence Springborg would discuss the governments commitment to genome sequencing research and ethics at the event.

Were excited that Minister Springborg can attend the forum to talk about why advances in genome sequencing present both opportunities and challenges for government, as well as for researchers and individuals, he said.

With genome sequencing now more affordable than ever, we envisage that it will bring major benefits for patients with cancer and also in rare and common inherited conditions.

As researchers uncover more about the human genome and its applications in medical practice, its clear that a robust ethical framework will be needed to guide clinical practice and research.

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Genetic research facility opens its doors to the public

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Why not embryonic stem cell
Why not embryonic stem cell ? In conversation with Dr Alok Sharma (MS, MCh.) Professor of Neurosurgery Head of Department, LTMG Hospital LTM Medical College, Sion, Mumbai. Stem Cell Therapy...

By: Neurogen Brain and Spine Institute

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Why not embryonic stem cell - Video

Recommendation and review posted by simmons

At the age of 70, many people are retired but Glenn Abbassi is still dashing round the world doing one of the most important jobs ever.

As a volunteer courier for bone marrow register Anthony Nolan, its her mission to travel thousands of miles transporting vital stem cells for seriously ill transplant patients.

So far, during seven years in her role, she has helped to save the lives of 264 people. She has travelled to four continents and more than 30 countries. She even spent last Christmas away from her family in China.

Speaking yesterday in support of a new Anthony Nolan campaign, she said: I wouldnt change it for the world. Every trip I embark on is as important as the next one.

Glenn, a former NHS complaints manager, explained how donated cells have to be with the recipient within 72 hours.

Getting back in time is a matter of life or death, she said.

The cells are used to treat a range of conditions, including cancer and blood disorders.

Glenns role is particularly poignant as her first husband Peter Davies was diagnosed with the blood disorder aplastic anaemia in 1977. He died three years later aged just 43.

She met her current husband Eddie, 68, a retired air conditioning engineer, a few years later when he flew to Britain from his homeland in Iran to donate his bone marrow to his brother.

They fell in love when Eddie lodged with her while his brother recovered.

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Amazing pensioner helps save 264 lives in 30 countries on four continents

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