AMSTERDAM, The Netherlands, December 6, 2012 /PRNewswire/ --

uniQure B.V., a leader in the field of human gene therapy, today announced the appointment of Hans Christian Rohde as Chief Commercial Officer.

Mr. Rohde joins uniQure from Basilea Pharmaceutica (SIX:BSLN) where from 2007 he was Chief Commercial Officer and member of the company's executive management committee with responsibility for global commercial operations, marketing, supply chain, medical affairs, pricing and market access.

"We are very pleased with Hans Christian's appointment," says Jrn Aldag, CEO of uniQure. "With the approval of Glybera we need to rapidly build and expand our commercial organization to enable the successful roll-out of Glybera and provide access to this crucial treatment to as many patients as possible. Hans Christian's experience and successful track-record in major biotechnology companies such as Basilea, Merck-Serono and Biogen Idec provide us with the necessary leadership qualities to successfully commercialize Glybera."

Hans Christian Rohde (M.Sc., MBA) has almost 25 years experience in commercial roles at leading biotechnology and pharmaceutical companies. Prior to Basilea Pharmaceutica, Mr. Rohde was Corporate Vice President, Head of Global Therapeutic Areas Reproductive Health and Endocrinology at Merck-Serono from 2003 until 2007. Before this he was responsible for international marketing and global market development at Biogen Idec. From 1992 until 2000, Mr Rohde held positions of increasing commercial responsibility at Novo Nordisk. Mr. Rohde started his career at Laboratoires Syntex.

About uniQure

uniQure is a world leader in the development ofhuman gene based therapies.uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform.This proprietary platform can be applied to a large number of rare(orphan) diseases caused by one faulty gene. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.

About Glybera

uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency, an orphan disease. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL enzyme in patients. This enzyme is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US, and obtained marketing authorisation from the European Commission for commercialization in the 27 EU countries in November, 2012, making it the first gene therapy to win approval in the Western world.

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uniQure Appoints Hans Christian Rohde Chief Commercial Officer

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Kell - ASD - 3 day after Stem Cell Treatment Part 2
For more information about ASD and Stem Cell Therapy, please visit worldstemcells.com 2012 World Stem Cells LLCFrom:WorldStem CellsViews:2 2ratingsTime:17:14More inScience Technology

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Making Stem Cell Thearpy Safer - Mayo Clinic
Mayo Clinic researchers have found a way to detect and eliminate potentially troublemaking stem cells to make stem cell therapy safer. Induced Pluripotent Stem cells, also known as iPS cells, are bioengineered from adult tissues to have properties of embryonic stem cells, which have the unlimited capacity to differentiate and grow into any desired types of cells, such as skin, brain, lung and heart cells. Study Author, Dr. Timothy Nelson provides and overview.From:mayoclinicViews:46 0ratingsTime:02:04More inScience Technology

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Making Stem Cell Thearpy Safer - Mayo Clinic - Video

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Stephen Quake, 2012 Lemelson-MIT Prize Winner
Dr. Stephen Quake, one of the world #39;s most prolific inventors, was recipient of the 2012 $500000 Lemelson-MIT Prize. An adventurer at heart, Quake relishes in the adrenaline that comes from mountain biking and ski mountaineering. It is that same rush he gets from exploring the unknown that drives him to consistently push scientific limits. Quake was recognized for his revolutionary work in drug discovery, genome analysis and personalized medicine.From:LemelsonMITProgramViews:1 0ratingsTime:04:37More inNonprofits Activism

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Stephen Quake, 2012 Lemelson-MIT Prize Winner - Video

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Kevin Krenitsky - How does a consumer find out about Foundation Medicine?
Kevin Krenitsky, Chief Operating Officer of Foundation Medicine sits down with Slone Partners at the Personalized Medicine Conference in Boston to discuss Foundation Medicine and their first clinical product, FoundationOne (www.foundationone.com). FoundationOne was released this year at ASCO (http and has since gained significant adoption in the industry. The 8th Annual Personalized Medicine Conference was hosted at Harvard Medical School by Partners Healthcare.From:SlonePartnersMediaViews:11 0ratingsTime:02:45More inScience Technology

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Kevin Krenitsky - How does a consumer find out about Foundation Medicine? - Video

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Kevin Krenitsky - Tell us about Foundation Medicine
Kevin Krenitsky, Chief Operating Officer of Foundation Medicine sits down with Slone Partners at the Personalized Medicine Conference in Boston to discuss Foundation Medicine and their first clinical product, FoundationOne (www.foundationone.com). FoundationOne was released this year at ASCO (http and has since gained significant adoption in the industry. The 8th Annual Personalized Medicine Conference was hosted at Harvard Medical School by Partners Healthcare.From:SlonePartnersMediaViews:9 0ratingsTime:03:34More inScience Technology

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Kevin Krenitsky - Tell us about Foundation Medicine - Video

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Protecting Your Shoulders and Staying Active after Spinal Cord Injury
If you push a wheelchair or use crutches for walking, your shoulders are working much harder than they were designed to do and can easily be damaged over time. How do you stay independent and active while avoiding shoulder pain and injury? This presentation by physical therapist Kristin Kaupang of Harborview Medical Center focuses on techniques and exercises for preserving and protecting your shoulders to minimize injury, decrease pain, increase strength, and maintain use of your shoulders for the long haul.From:UWSpinalCordInjuryViews:3 0ratingsTime:01:01:55More inScience Technology

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Protecting Your Shoulders and Staying Active after Spinal Cord Injury - Video

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World Stem Cell Summit - Terzic
The World Stem Cell Summit, December 3-5 in West Palm Beach, Fla. unites internationally renowned scientists, clinicians, students, policymakers, regulators, patient advocates, and philanthropists to share knowledge and advance collaborations in the stem cell and regenerative medicine community. The meeting includes in-depth programming and more than 150 renowned international speakers. In this video, Dr. Andre Terzic, director of the Mayo Clinic Center for Regenerative Medicine addresses the importance of the summit and the emerging field of regenerative medicine.From:mayoclinicViews:6 0ratingsTime:05:34More inScience Technology

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The World Stem Cell Summit - Dr. Yaszemski
The World Stem Cell Summit, December 3-5 in West Palm Beach, Fla. unites internationally renowned scientists, clinicians, students, policymakers, regulators, patient advocates, and philanthropists to share knowledge and advance collaborations in the stem cell and regenerative medicine community. The meeting includes in-depth programming and more than 150 renowned international speakers, including leaders from Mayo Clinic #39;s Center for Regenerative Medicine. Among them is Mayo Clinic Dr. Michael Yaszemski. In this video he talks about the summit, the emerging field of regenerative medicine and his unique military perspective.From:mayoclinicViews:5 1ratingsTime:04:16More inScience Technology

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World Stem Cell Summit - Windebank
The World Stem Cell Summit, December 3-5 in West Palm Beach, Fla. unites internationally renowned scientists, clinicians, students, policymakers, regulators, patient advocates, and philanthropists to share knowledge and advance collaborations in the stem cell and regenerative medicine community. The meeting includes in-depth programming and more than 150 renowned international speakers, including leaders from Mayo Clinic #39;s Center for Regenerative Medicine. In this video, Dr. Anthony Windebank talks about the emerging field of regenerative medicine.From:mayoclinicViews:8 0ratingsTime:03:39More inScience Technology

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ScienceDaily (Dec. 3, 2012) Researchers have derived dopaminergic neurons from bone marrow stem cells in monkeys.

Parkinson's disease is a degenerative disorder of the central nervous system that is characterized by tremors, rigidity, slowness of movement, and difficulty walking. It is caused by loss of the neurons that produce the neurotransmitter dopamine (known as dopaminergic neurons). One of the primary goals in Parkinson's disease research is to develop a replacement for dopaminergic neurons.

In a new study, researchers led by Takuya Hayashi at the RIKEN Center for Molecular Imaging Science in Kobe, Japan, derived dopaminergic neurons from bone marrow stem cells in monkeys. The cells were retrieved during a standard bone marrow aspiration and then treated with growth factors that directed the stem cells to become dopaminergic neurons. The monkeys that donated the stem cells were treated with a chemical to induce Parkinson's disease and then received a transplant of the new dopaminergic neurons that had been derived from their own bone marrow stem cells. Monkeys that received the transplant showed significant improvement in motor defects.

This study demonstrates that dopaminergic neurons derived from adult bone marrow stem cells can be safely used to improve motor function in Parkinson's disease in monkeys.

The research is published in the Journal of Clinical Investigation.

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Note: Materials may be edited for content and length. For further information, please contact the source cited above.

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Stem cell-derived dopaminergic neurons rescue motor defects in Parkinsonian monkeys

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BOCA RATON, Fla.--(BUSINESS WIRE)--

Akron Biotechnology, LLCs Quality Management System was registered on November 30th, 2012 on the requirements of ISO 9001:2008 standards by BSI Group America Inc. This ISO Certification emphasizes Akron Biotechs commitment to quality and meeting their customers needs and expectations. It covers all relevant functions of the Company: development, production, storage and distribution of Akron Biotechs products, i.e. cryopreservation media, recombinant proteins, media supplements, scaffolds and cell culture systems.

"This is an important step in our commitment to quality standards that will continue to enhance our products and services. We are looking forward to building upon this base towards a stronger foundation of excellence and compliance as we develop innovative products within our pipeline." said Claudia Zylberberg, PhD, Akrons President and CEO. She further stated We are committed to providing our customers with the unique ability to seamlessly transition from R&D to pre-clinical and clinical development with minimal change control in order for them to focus on their clinical outcome.

About ISO 9001:2008 - Quality Management System

ISO 9001:2008 is one of the most widely used management systems related to quality standards and ensuring that a company is meeting the needs of customers and regulatory requirements. Third party certification bodies (e.g. BSI Group America, Inc) provide independent confirmation that an organization has met the requirements of ISO 9001:2008.

About Akron Biotech

As a global supplier, Akron Biotech (www.akronbiotech.com) manufactures and distributes components and raw materials for cell therapy discovery, development and commercialization, meeting the industrys needs worldwide. The Companys unique business model emphasizes flexibility, quality and unparalleled service from research and development through clinical trial to market authorization and commercial product.

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Akron Biotech, a Leading Global Supplier for the Cell Therapy Industry, Receives ISO 9001:2008 Certification

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WALTHAM, Mass.--(BUSINESS WIRE)--

Histogenics Corp., a biologics company that combines cell therapy and tissue engineering technologies to develop highly innovative products primarily for orthopaedic tissue repair and regeneration, today announced the appointment of Kevin L. Rakin to its Board of Directors. Mr. Rakin has over 20 years of experience in the biotechnology industry and most recently served as President of Regenerative Medicine at Shire plc.

We are very pleased to welcome Kevin to the Histogenics Board of Directors. His broad experience in biotechnology and regenerative medicine over a 20 year career will be extremely valuable during this exciting time for the company, said Patrick ODonnell, President and Chief Executive Officer of Histogenics. We believe that Kevins experience with regenerative medicine treatments and understanding of niche markets will help us advance the clinical development program of NeoCart and prepare it for commercialization.

Kevin Rakin was most recently the President of Regenerative Medicine at Shire plc, a leading specialty biopharmaceutical company. Prior to joining Shire, Mr. Rakin served as the Chairman and CEO of Advanced BioHealing (ABH) from 2007 until its acquisition by Shire for $750 million in June 2011. Before this, he served as the interim CEO of ABH and executive-in-residence at Canaan Partners. Previously, he was a co-founder, President and Chief Executive Officer of Genaissance Pharmaceuticals Inc., a publicly held pharmacogenomics company, until its merger with Clinical Data Inc. in October 2005.

He currently serves on the executive committee for Connecticut United for Research Excellence (CURE), Connecticuts bioscience cluster. He has previously served as a board member for Ipsogen SA, Vion Pharmaceuticals Inc., OMRIX Biopharmaceuticals Inc. and Clinical Data Inc. Mr. Rakin holds an M.B.A. from Columbia University and received his graduate and undergraduate degrees in Commerce from the University of Cape Town, South Africa.

About Histogenics

Histogenics is a leading regenerative medicine company that combines cell therapy and tissue engineering technologies to develop highly innovative products for tissue repair and regeneration. In May of 2011, Histogenics acquired Israeli cell-therapy company ProChon BioTech. Histogenics flagship products focus on the treatment of active patients suffering from articular cartilage derived pain and immobility. The Company takes an interdisciplinary approach to engineering neocartilage that looks, acts and lasts like hyaline cartilage. It is developing new treatments for sports injuries and other orthopedic conditions, where demand is growing for long-term alternatives to joint replacement. Histogenics has successfully completed Phase 1 and Phase 2 clinical trials in which the NeoCart autologous tissue implants effectiveness is compared to that of standard microfracture surgery. Based in Waltham, Massachusetts, the company is privately held. For more information, visitwww.histogenics.com.

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Kevin Rakin Appointed to the Histogenics Board of Directors

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NEW YORK, Dec. 3, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS) ("NeoStem" or the "Company"), an emerging leader in the fast growing cell therapy market, today announced that the Company and its subsidiary, Progenitor Cell Therapy ("PCT"), will present at multiple conferences in December.

World Stem Cell Summit

RedChip Small-Cap Equities Virtual Conference

Cell Therapy Manufacturing (Informa Life Sciences' 3rd Annual VIC Congress 2012)

American Society of Hematology Annual Meeting and Exposition ("ASH")

NeoStem's abstract on VSELTM Technology titled "A Lin-CD45-CD34+ Population of Extracellular Vesicles in Human Blood That Mimics Very Small Embryonic-Like Stem Cells (VSELs) by Flow Cytometry" has been selected for publication online in December in conjunction with the ASH Annual Meeting and Exposition, December 8-11, 2012. The abstract will be available at http://bloodjournal.hematologylibrary.org/site/misc/ASH_Meeting_Abstracts_Info.xhtml.

About NeoStem, Inc.

NeoStem, continues to develop and build on its core capabilities in cell therapy, capitalizing on the paradigm shift occurring in medicine. We anticipate that cell therapy will have a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy industry. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, PCT, with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe that, with our expertise and research capabilities and collaborations, we will achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cell product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert to create a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a substantial intellectual property estate, we are well positioned to succeed.

For more information, please visit http://www.neostem.com.

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NeoStem to Present at Multiple Conferences in December

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GULF SHORES, Ala., Dec. 4, 2012 /PRNewswire/ --More than 75 percent of amyotrophic lateral sclerosis (ALS) patients who received neural reprogrammed stem cell therapy have shown a positive response to the procedure. The groundbreaking technique was introduced to the United States this year by Precision StemCell (http://www.precisionstemcell.com), an outpatient imaging and image-guided treatment facility located in Gulf Shores, Ala.

The procedure is performed by Dr. Jason R. Williams, a board-certified radiologist with extensive training in image-guided procedures. Under his care, 14 out of 18 patients diagnosed with ALS, also known as Lou Gehrig's disease, have shown signs of recovery.

"The improvements are mild, with patients reporting improved movement, breathing and speech, but we still have a long way to go," Dr. Williams stated. "Only time will tell how this therapy will affect the patients' long-term prognosis."

In Precision StemCell's neural reprogrammed stem cell therapy, fat-derived stem cells are injected into the spine of the patient. Dr. Williams uses a drug called selegeline, which has been shown to be a pre-inducer of adipose-derived stem cells into neural-like cells. Dr. Williams contends that the therapy is probably one of the largest advances seen in ALS therapy. "Before we started this therapy, I would have been happy just to see the progression of the disease halted, but to see some actual improvement, that was just shocking," he said

The first patient treated with the technique was Frank Orgel who continues to see improvement since his first treatment seven months ago. Eight years ago, Orgel's quality of life had declined to the point that he could not move his left arm or leg, walk or even stand on his own. The therapy has allowed Orgel to stand without assistance, and he continues to work with a physical therapist to regain the ability to walk. Another patient, Dexter Johnson, previously walked with a cane. After the treatment, Johnson has been able to walk without his cane for the majority of the time and he has been able to walk at a much faster pace.

The Precision StemCell center focuses on advanced imaging techniques, which include a 3T Open MRI, a low-dose 64-Slice CT Scanner, ultrasound and fluoroscopy, also known as real-time x-ray. The center is headed by Dr. Williams, who specializes in image-guided procedures, had already been performing magnetic resonance imaging (MRI), computed tomography (CT), ultrasound and fluoroscopy-guided stem cell injections for joint and orthopedic conditions.

In addition to their current work on advanced stem cell harvesting and processing with image-guided stem cell injections, Precision StemCell staff are planning to conduct further research so as to develop even more advanced techniques such as adding gene therapy to the current neural reprogramming platform. "Our therapy techniques not only hold promise for ALS patients, but also for people with other neural-related conditions such as Parkinson's and spinal cord injuries," said Dr. Williams. "There are several candidate genes that we plan to add to the adipose-derived stem cells and study in the mouse model. Though we are happy with our initial progress, our goal is to develop an effective cure."

About Precision StemCell

Located in Gulf Shores, Alabama, Precision StemCell conducts stem cell procedures using advanced imaging techniques, which include an Open 3T MRI, a low-dose 64-Slice CT scanner, ultrasound, and real time x-ray (fluoroscopy). Headed by Jason Williams, MD, a board-certified radiologist with extensive training in image-guided procedures, the facility performs advanced stem cell harvesting and processing with image-guided stem cell injections.

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Precision StemCell's Neural Reprogrammed Stem Cell Therapy Yields Better-Than-Expected Results for ALS Patients

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(DGAP-Media / 04.12.2012 / 10:15) Munich, Bielefeld, Heidelberg (Germany), SIRION Biotech and PlasmidFactory agreed to actively promote adeno-associated virus (AAV) vectors for advanced preclinical research. Dr. Oliver Mueller, University of Heidelberg, will serve as scientific advisor. AAV vectors have been identified as most promising gene delivery candidates for curative therapy of gene defects that need long-term treatment. It is estimated that over 4000 human diseases are caused by single gene defects. Besides acting as highly versatile gene delivery agents they also serve for functional genomic studies. To date 44 clinical trials are being conducted with AAV for diseases like Parkinson, Alzheimer or Stage IV Gastric cancer. The first AAV vector-based gene therapy for lipoprotein lipase deficiency (LPLD) has been approved in Europe for Human Use just a few weeks ago. Generating AAV, especially for use in preclinical and clinical applications requires a mix of skills: plasmid DNA in reproducible and certified quality both in research and clinical grades, solid experience in generating AAV vectors for preclinical use and the ability to generate entire cell models according to desired specifications. This week's agreement between PlasmidFactory and SIRION Biotech allows for commercial supplies of highly innovative AAV vectors for preclinical research and up to clinical phase III studies. Within just 8 weeks SIRION Biotech is able to supply research quantities for in vivo studies in small animals. Working with AAV vectors for many more clinical trials is now made a realistic option for researchers worldwide. About SIRION BIOTECH, PlasmidFactory and PD Dr. Oliver Mller SIRION Biotech was founded in 2006 in Munich and has sites in Tokyo and New Hampshire. It stands for sophisticated cell modelling and viral vector platforms. These enable much improved target identification and compound screening in the drug, the food & cosmetic industries. Its technology has over the years been validated in more than 250 commercial projects with more than 70 academic and industrial organizations worldwide. As a result, primary cell immortalizations are being technically advanced, so is the recombinant virus vector construction serving for gene therapy applications and novel vaccines. PlasmidFactory is a biopharmaceutical company, founded in Bielefeld in 2000. Today the company is formed by a young team of biologists, chemists, engineers and technicians and is Europe's leading contract manufacturer of plasmid DNA. The company produces clients' plasmids according to their requirements (incl. GMP use) in modern laboratories with high quality standards. PlasmidFactory holds the worldwide exclusive license from DKFZ for manufacturing, distribution and use of the Helper & Packaging Plasmids from the pDG and pDP family, respectively1. Dr. Oliver Mueller leads a team at the Dept. of Internal Medicine III at the University of Heidelberg whose aim is to develop gene therapies for hereditary heart diseases. The department's research is addressing chronic diseases by close interaction of medical and biology basic research. Contact: SIRION BIOTECH GmbH Dr. Christian Thirion Am Klopferspitz 19 D-82152 Martinsried Tel.: +49-89-700 961 99-15 eMail: Thirion@Sirion-Biotech.com http://www.Sirion-Biotech.com PlasmidFactory GmbH & Co. KG Dr. Martin Schleef Meisenstr. 96 D-33607 Bielefeld Tel.: +49-521-299 7350 eMail: info@PlasmidFactory.com http://www.PlasmidFactory.com 1References: [1] Grimm et al. (1998), Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors, Human Gene Therapy, Vol. 9, 2745-2760. [2] Raake PWJ, Schlegel P, Ksienzyk J, Reinkober J, Barthelmes J, Schinkel S, Pleger S, Mier W, Haberkorn U, Katus HA, Koch WJ, Most P, Muller OJ. AAV6.ARKct cardiac gene therapy ameliorates cardiac function and normalizes the catecholaminergic axis in a clinically relevant large animal heart failure model. Europ Heart J. Jan 12. (2012) [Epub ahead of print] [3] Grimm et al. (2003), Helper Virus-Free, Optically Controllable, and Two-Plasmid-Based Production of Adeno-associated Virus Vectors of Serotypes 1 to 6, Molecular Therapy, Vol. 7, 839-850. [4] Kronenberg et al. (2005), A Conformational Change in the Adeno-Associated Virus Type 2 Capsid Leads to the Exposure of Hidden VP1 N Termini, Journal of Virology, Vol. 79, 5296-5303. [5] Moullier, P. and Snyder, R.O. (2008), International efforts for recombinant adenoassociated viral vector reference standards, Molecular Therapy, Vol. 16, 1185-1188 [6] AAV-DNA helper sequences EP 0 934 423 End of Media Release =-------------------------------------------------------------------- Issuer: Sirion Biotech GmbH Key word(s): Health 04.12.2012 Dissemination of a Press Release, transmitted by DGAP - a company of EquityStory AG. The issuer is solely responsible for the content of this announcement. DGAP's Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases. Media archive at http://www.dgap-medientreff.de and http://www.dgap.de =-------------------------------------------------------------------- 195744 04.12.2012

(END) Dow Jones Newswires

December 04, 2012 04:15 ET (09:15 GMT)

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Sirion Biotech GmbH: Adeno-associated virus now easily available for research and preclinical studies

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Institutes to Share Resources to Identify Potential Compounds Targeting TDP-43 Form of ALS

CAMBRIDGE, MA and SAN FRANCISCO, Dec. 4, 2012 /PRNewswire-USNewswire/ -- The ALS Therapy Development Institute (ALS TDI) and the Gladstone Institutes today announced the formation of a research collaboration to speed the discovery of potential treatments for ALS through the preclinical drug development process.

(Logo: http://photos.prnewswire.com/prnh/20110204/DC42625LOGO)

"We are thrilled about the potential this collaboration holds to accelerate ALS therapeutic development," said Steve Perrin, PhD, CEO and Chief Scientific Officer at ALS TDI. "Both our organizations have unique infrastructures, and by linking them this way, we may be able to advance potential treatments faster than before."

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a neurodegenerative disease that leads to paralysisand eventually deathdue to the loss of motor neurons in the spinal cord and brain. About 30,000 people in the United States live with the disease today, and the global population of ALS patients is approximately 400,000. Approximately 5,000 new cases of ALS are diagnosed in the United States each year, and there is no known cure or treatment to halt or reverse the disease. The average patient survives only two to five years following their diagnosis.

Under this new agreement, which starts immediately, Gladstone will evaluate potential pharmaceutical compounds using a human model of ALS. Gladstone generated the model by transforming skin cells from ALS patients into stem cells, known as induced pluripotent stem cells (iPS cells), and then programming them into neurons. The technique builds on a discovery for which Shinya Yamanaka, MD, PhD, a Gladstone senior investigator, won the 2012 Nobel Prize in Physiology or Medicine.

This particular iPS-based ALS model includes a gene mutation that produces TDP-43, a protein commonly found in most forms of ALS. Promising drug compounds that pass the initial evaluation process at Gladstone will be fast-tracked for pre-clinical testing at ALS TDI, which will assess the compounds for activity and efficacy in various mouse models of human neurodegeneration.

"We hope our human model of ALS will help us to move quickly and effectively to identify promising therapeutic candidates for ALS," said Gladstone Senior Investigator Steve Finkbeiner, MD, PhD, who is also a professor of neurology and physiology at the University of California, San Francisco, with which Gladstone is affiliated. "The strong evidence that abnormal TDP-43 protein is involved in the development of ALS, coupled with models that may replicate ALS more faithfully than other tools, may speed development of therapies for the thousands of individuals diagnosed with this devastating disease."

The collaborators will conduct a joint webinar to discuss the project plan in depth in January 2013. To register, visit http://www.als.net/webinars.

About the ALS Therapy Development Institute (ALS TDI)

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ALS Therapy Development Institute And Gladstone Institutes Collaborate To Discover Potential ALS Treatments

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Lawrence LeBlond for redOrbit.com Your Universe Online

Its an undeniable fact that many teenagers are going to experiment with alcohol before they are mentally ready enough to do so. Now, a new study may help explain why some of these teenagers are more prone to continue drinking alcohol after trying it than others are.

A study, led by researchers at Kings College Londons Institute of Psychiatry (IoP), provides the most detailed understanding yet of the brain process involved in alcohol abuse. The research team recently discovered a geneRASGRF-2which is one of many already suggested being associated with binge drinking.

Publishing their work in the journal Proceedings of the National Academy of Sciences (PNAS), the team found that animals lacking the gene had far less desire for alcohol than those who did have it. Adding to that finding, brain scans of 663 teenage boys showed that those with the human version of the gene had heightened dopamine responses in tests.

Alcohol, as well as some addictive drugs, activates the dopamine system in the brain which is responsible for feelings of pleasure and reward. Recent studies on RASGRF-2 in mice showed that the gene is a risk factor for alcohol abuse; yet, the exact mechanism involved in the process has remained a mystery until now.

During a task designed to make the test subjects anticipate a reward, the 14-year-old boys had more activity in an area of the brain called the ventral striatum, an area known to be involved with dopamine release. When the boys were contacted again at age 16 and asked about their drinking habits, the team found the boys with the RASGRF-2 variation drank more frequently than those who didnt have the gene.

Lead researcher Professor Gunter Schuman acknowledged that his teams findings do not necessarily prove that the gene variation causes binge drinking, but noted a variety of environmental factors along with several genes could be the right ingredients to contribute to the issue. He does believe, however, that his teams findings help shed light on why some people appear to be vulnerable to the allure of alcohol.

Schuman said the gene appears to regulate how rewarding alcohol is for some people.

People seek out situations which fulfill their sense of reward and make them happy, so if your brain is wired to find alcohol rewarding, you will seek it out. We now understand the chain of action: how our genes shape this function in our brains and how that, in turn, leads to human behavior, he said in a statement.

We found that the RASGRF-2 gene plays a crucial role in controlling how alcohol stimulates the brain to release dopamine, and hence trigger the feeling of reward. So, if people have a genetic variation of the RASGRF-2 gene, alcohol gives them a stronger sense of reward, making them more likely to be heavy drinkers, Schuman continued.

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Gene Plays A Role In Binge Drinking In Young Teenagers

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LONDON (Reuters) - Scientists have unpicked the brain processes involved in teenage alcohol abuse and say their findings help explain why some young people have more of a tendency to binge drink.

A study published in the Proceedings of National Academy of Sciences (PNAS) journal found that a gene known as RASGRF-2 plays a crucial role in controlling how alcohol stimulates the brain to release dopamine, triggering feelings of reward.

"If people have a genetic variation of the RASGRF-2 gene, alcohol gives them a stronger sense of reward, making them more likely to be heavy drinkers," said Gunter Schumann, who led the study at King's College London's Institute of Psychiatry.

Alcohol and other addictive drugs activate the brain's dopamine systems, which induces feelings of pleasure and reward.

Worldwide, some 2.5 million people die each year from the harmful use of alcohol, accounting for about 3.8 percent of all deaths, according to the World Health Organisation.

Recent studies also carried out by scientists at the IoP have found that RASGRF-2 is a risk gene for alcohol abuse, but until now the mechanism involved in the process was not clear.

For this study, scientists initially looked at mice who had been modified to have the RASGRF2 gene removed, to see how they reacted to alcohol. They found the lack of RASGRF-2 was linked to a significant reduction in alcohol-seeking activity.

They also discovered that when the mice did consume alcohol, the absence of RASGRF-2 reduced the activity of dopamine-releasing neurons in a region of the brain called the ventral tegmental area (VTA) - preventing the brain from releasing dopamine and limiting any sense of reward.

The team then analyzed brain scans of 663 14-year old boys and found that when they were anticipating a reward in a mental test, those with genetic variations to the RASGRF2 gene had more activity in an area of the brain closely linked to the VTA and also involved in dopamine release.

This suggests people with a genetic variation on the RASGRF-2 gene release more dopamine when anticipating a reward, and hence derive more pleasure from it, the scientists said.

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Scientists find gene link to teenage binge drinking

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FIRST PERSON | The Supreme Court announced on Nov. 30 that it plans to rule on the issue of gene patents. This stems from a case in 2010, when the American Civil Liberties Union (ACLU) took the drug research company Myriad Labs to court over its patent on the BRCA gene. The ACLU claims that naturally occurring genes should not be patented. Myriad Labs disagrees, stating that it will not be financially viable to conduct important medical research without the ability to patent.

To patent or not

CBS News reported that the Supreme Court plans to hear the case in early spring 2013. This will finally lay to rest the question as to whether human genes are patentable. It is not a simple question. Right now, only Myriad Labs can test or perform medical research on the BRCA genes. Researchers wishing to test the BRCA gene sequence must pay royalties to Myriad Labs.

BRCA genes

The BRCA genes are an important sequence from a research standpoint. Certain mutations involving these genes leave women at high risk for breast, ovarian, and other cancers. The BRCA1 and BRCA2 gene mutations are at the heart of this legal battle. Testing for BRCA1 or BRCA2 is extremely expensive. Because Myriad Labs holds the patent, no other company can design a less expensive test.

Testing

I had genetic testing done because of my family history with breast cancer. It involved genetic counseling and a blood draw. Testing from Myriad Labs cost $5,000. Insurance paid for some of it, but I still had a significant amount to pay out of pocket. Women who receive Medicare, Medicaid, or those without insurance may not be able to afford the test -- leaving them without access to potentially life-saving medical information.

If you are BRCA positive, your risks for cancer skyrocket. Important decisions about your health care, including preventative surgical procedures, must be made. Sharing your BRCA status with immediate family members may impact important life decisions such as deciding to have children. This is why the ACLU sued.

Genes should not have patents

I had a diagnosis of breast cancer before undergoing genetic testing. It was important for me to share the test results with my family. Fortunately, my cancer is not BRCA1 or 2 related. This does not rule out my cancer being genetic. Here is the problem with Myriad Labs owning the patent on the gene: They are not looking for new mutations and no one else can undertake this important research without paying royalties to Myriad.

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Supreme Court to Decide If Gene Patents Are Legal: It's About Time

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Featured Article Academic Journal Main Category: Alcohol / Addiction / Illegal Drugs Also Included In: Genetics;Pediatrics / Children's Health Article Date: 04 Dec 2012 - 3:00 PST

Current ratings for: Gene May Prime Teens To Binge Drink

5 (1 votes)

The study, led by King's College London's Institute of Psychiatry (IoP) is published online this week in the Proceedings of National Academy of Sciences, PNAS.

But what has become more common in this age group is binge drinking: consuming large amounts of alcohol in a short space of time.

Studies show teens's alcohol consumption has gone up from 6 units a week in 1994 to 13 in 2007.

The authors note that every year in the UK, around 5,000 teenagers are hospitalized because of drink.

Apart from the obvious problems such as increased anti-social and risk-taking behavior (for example drunk driving and unsafe sex), alcohol abuse in teenagers has also been linked to poor brain development and long term health problems.

Several genes have been linked to risk for alcohol abuse, including RASGRF-2, identified in recent studies from King's IoP.

But this latest study is the first to suggest the underlying mechanism through which the gene stimulates the brain to release dopamine.

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Gene May Prime Teens To Binge Drink

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Why do some people binge drink? It may be in their genes.

Researchers from King's College London's Institute of Psychiatry have previously discovered that the RASGRF2 gene is linked to alcohol abuse. A study published in the Proceedings of the National Academy of Sciences on Dec. 3 now shows how that gene affects alcoholics.

The RASGRF-2 gene works to control how the brain releases dopamine, a chemical neurotransmitter that makes people feel pleasure and reward. Alcohol and other addictive substances stimulate the brain's dopamine system.

"So, if people have a genetic variation of the RASGRF-2 gene, alcohol gives them a stronger sense of reward, making them more likely to be heavy drinkers," lead author Gunter Schumann, professor of biological psychiatry, said in a press release.

One out of six people in the United States has a drinking problem, the National Institutes of Health report.

In 2009, 10.4 million people between the ages of 12 and 20 admitted to drinking more than a few sips of alcohol, according to the National Institute of Alcohol Abuse and Alcoholism. By age 15, 70 percent of teens have had at least one drink. More than 190,000 people under the age of 21 went to the emergency room for an alcohol-related injury in 2008 alone. Five thousand people under 21 die each year from alcohol-related car crashes, homicides, suicides, alcohol poisoning, and other injuries such as falls, burns, and drowning.

People who are at risk of becoming alcoholics are men who have 15 or more drinks a week, or women who have 12 or more drinks a week, according to the National Institutes of Health. Anyone who has five or more drinks per occasion at least once a week is also at risk. On average, while young adults drink less often than adults they drink five drinks on a single occasion.

Young adults under peer pressure, people with history of depression and other mental disorders, easy access of alcohol, low self-esteem, problems with relationships, stressful lifestyles and those who live in a culture where drinking is common and accepted have a higher chance of developing alcoholism.

The study first looked at mice without the RASGRF-2 gene to see how alcohol affected them. They found these mice had significantly lowered alcohol-seeking behavior because the lack of the gene partially blocked their brains from releasing dopamine -- the reward sensation -- from an area of the brain called the ventral tegmental area (VTA).

Researchers then scanned the brains of 663 14-year-old boys who had not been exposed to large amounts of alcohol. Individuals who had a certain genetic variation of the RASGRF2 gene had more activation in their VTA region, meaning more dopamine was released when they were looking forward to a reward from a cognitive task. This meant that their brains released more dopamine when they were about to receive something good, giving them more happiness from that experience.

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Gene variation may make a person more likely to binge drink

Recommendation and review posted by Bethany Smith

Why do some people binge drink? It may be in their genes.

Researchers from King's College London's Institute of Psychiatry have previously discovered that the RASGRF2 gene is linked to alcohol abuse. A study published in the Proceedings of the National Academy of Sciences on Dec. 3 now shows how that gene affects alcoholics.

The RASGRF-2 gene works to control how the brain releases dopamine, a chemical neurotransmitter that makes people feel pleasure and reward. Alcohol and other addictive substances stimulate the brain's dopamine system.

"So, if people have a genetic variation of the RASGRF-2 gene, alcohol gives them a stronger sense of reward, making them more likely to be heavy drinkers," lead author Gunter Schumann, professor of biological psychiatry, said in a press release.

One out of six people in the United States has a drinking problem, the National Institutes of Health report.

In 2009, 10.4 million people between the ages of 12 and 20 admitted to drinking more than a few sips of alcohol, according to the National Institute of Alcohol Abuse and Alcoholism. By age 15, 70 percent of teens have had at least one drink. More than 190,000 people under the age of 21 went to the emergency room for an alcohol-related injury in 2008 alone. Five thousand people under 21 die each year from alcohol-related car crashes, homicides, suicides, alcohol poisoning, and other injuries such as falls, burns, and drowning.

People who are at risk of becoming alcoholics are men who have 15 or more drinks a week, or women who have 12 or more drinks a week, according to the National Institutes of Health. Anyone who has five or more drinks per occasion at least once a week is also at risk. On average, while young adults drink less often than adults they drink five drinks on a single occasion.

Young adults under peer pressure, people with history of depression and other mental disorders, easy access of alcohol, low self-esteem, problems with relationships, stressful lifestyles and those who live in a culture where drinking is common and accepted have a higher chance of developing alcoholism.

The study first looked at mice without the RASGRF-2 gene to see how alcohol affected them. They found these mice had significantly lowered alcohol-seeking behavior because the lack of the gene partially blocked their brains from releasing dopamine -- the reward sensation -- from an area of the brain called the ventral tegmental area (VTA).

Researchers then scanned the brains of 663 14-year-old boys who had not been exposed to large amounts of alcohol. Individuals who had a certain genetic variation of the RASGRF2 gene had more activation in their VTA region, meaning more dopamine was released when they were looking forward to a reward from a cognitive task. This meant that their brains released more dopamine when they were about to receive something good, giving them more happiness from that experience.

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Gene variation may make a person more likely to drink alcohol more

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Genetic Engineering Magic!
Have fun everyone! All clips are courtesy of HistoricClips.com!From:MrPenflipViews:0 0ratingsTime:03:12More inPeople Blogs

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Genetic Engineering Magic! - Video

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The Truth About Genetic Engineering
This is the truth about genetic engineering, Genetic modification, and genetic enhancement.From:seaweed603Views:0 0ratingsTime:11:47More inNews Politics

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The Truth About Genetic Engineering - Video

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