Volume 13.10 | Mar 19

Issue 13.10, March 19, 2012
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Stem Cells Hint at Potential Treatment for Huntington’s Disease
Researchers have shown that the severe motor deficits observed in a mouse model of Huntington’s can be corrected by implanting lab made GABA neurons. [Press release from the University of Wisconsin-Madison discussing online prepublication in Cell Stem Cell] Press Release | Abstract

Read White Paper: Increase Success Rate Of Compounds In Toxicity Testing

PUBLICATIONS (Ranked by Impact Factor of the Journal)

Wild-Type Microglia Arrest Pathology in a Mouse Model of Rett Syndrome
Researchers found that transplantation of wild-type bone marrow into irradiation-conditioned Mecp2-null hosts resulted in engraftment of brain parenchyma by bone-marrow-derived myeloid cells of microglial phenotype, and arrest of disease development. [Nature] Abstract | Press Release

Prevention of Hepatocellular Adenoma and Correction of Metabolic Abnormalities in Murine Glycogen Storage Disease Type Ia by Gene Therapy
Researchers show that gene therapy mediated by adeno-associated virus-G6PC promoter/enhancer, maintains efficacy for at least 70-90 weeks for mice expressing more than 3% of wild type hepatic glucose-6-phosphatase-α activity. [Hepatology] Abstract

Correcting Human Mitochondrial Mutations with Targeted RNA Import
Here, scientists report that a 20-ribonucleotide stem-loop sequence from the H1 RNA, the RNA component of the human RNase P enzyme, appended to a nonimported RNA directs the import of the resultant RNA fusion transcript into human mitochondria. [Proc Natl Acad Sci USA] Abstract | Press Release

Mesenchymal Stem Cells: A Double-Edged Sword in Regulating Immune Responses
Previous studies have shown that mesenchymal stem cells (MSCs) can become highly immunosuppressive upon stimulation by inflammatory cytokines, an effect exerted through the concerted action of chemokines and nitric oxide. Here, researchers show that MSCs can also enhance immune responses. [Cell Death Differ] Abstract

Treatment of a Mouse Model of Spinal Cord Injury by Transplantation of Human iPS Cell-Derived Long-Term Self-Renewing Neuroepithelial-Like Stem Cells
Here, researchers show that transplanted long-term, self-renewing, neuroepithelial-like stem cells from human induced pluripotent stem (iPS) cells differentiate into neural lineages in the mouse model of spinal cord injury and promote functional recovery of hind limb motor function. [Stem Cells] Abstract

Neurotrophic Bone Marrow Cellular Nests Prevent Spinal Motoneuron Degeneration in Amyotrophic Lateral Sclerosis Patients: A Pilot Safety Study
The objectives of this study were to assess the safety of intraspinal infusion of autologous bone marrow mononuclear cells and, ultimately, to look for histopathological signs of cellular neurotrophism in amyotrophic lateral sclerosis patients. [Stem Cells] Abstract

Lumbar Intraspinal Injection of Neural Stem Cells in Patients with ALS: Results of a Phase I Trial in 12 Patients
Here researchers report the results of an ongoing Phase I trial of intraspinal injections of fetal-derived neural stems cells in patients with Amyotrophic Lateral Sclerosis (ALS). [Stem Cells] Abstract

In Vivo Evaluation of a Cancer Therapy Strategy Combining HSV1716-Mediated Oncolysis with Gene Transfer and Targeted Radiotherapy
Researchers used engineered herpes simplex virus 1 deletion mutant HSV1716 which conveys the noradrenaline transporter (NAT) gene as a dual cell lysis-gene delivery vehicle for targeting the NAT transgene to human tumor xenografts in vivo. [J Nucl Med] Abstract

Atopic Keratinocytes Induce Increased Neurite Outgrowth in a Coculture Model of Porcine Dorsal Root Ganglia Neurons and Human Skin Cells
Using an in vitro coculture system, allowing a spatially compartmented culture of somata from porcine dorsal root ganglion neurons and human primary skin cells, researchers investigated the influence of dermal fibroblasts and keratinocytes on neurite outgrowth. [J Invest Dermatol] Abstract

Cell Therapy for Skin Wound Using Fibroblast Encapsulated Poly(Ethylene Glycol)-Poly(L-Alanine) Thermogel
As a new application of a thermogel, a poly(ethylene glycol)-b-poly(L-alanine) gel encapsulating fibroblasts was investigated for wound healing. [Biomacromolecules] Abstract

X-Linked Inhibitor of Apoptosis Protein-Mediated Attenuation of Apoptosis, Using a Novel Cardiac-Enhanced Adeno-Associated Viral Vector
These studies demonstrate the potential benefit of X-linked inhibitor of apoptosis protein to correct heart failure after highly efficient delivery to the heart with the rationally designed SASTG adeno-associated viral vector. [Hum Gene Ther] Abstract

Targeting Bone Remodeling by Isoflavone and 3,3′-Diindolylmethane in the Context of Prostate Cancer Bone Metastasis
In this study, researchers found that prostate cancer cells could stimulate differentiation of osteoclasts and osteoblasts through the up-regulation of RANKL, RUNX2 and osteopontin, promoting bone remodeling. [PLoS One] Abstract

Blood-Derived Human iPS Cells Generate Optic Vesicle-Like Structures with the Capacity to Form Retinal Laminae and Develop Synapses
Researchers sought to determine if human induced pluripotent stem (iPS) cells derived from blood could produce optic vesicle-like structures with the capacity to stratify and express markers of intercellular communication. [Invest Ophthalmol Vis Sci] Abstract | Press Release

A Model for Personalized In Vivo Analysis of Human Immune Responsiveness
Here, researchers demonstrated robust immune reconstitution in mice with hematopoietic stem cells aspirated from bone marrow of adults with type 1 diabetes and healthy control volunteers. [Sci Transl Med]
Abstract | Press Release

Prospective Trial of Adipose-Derived Regenerative Cell (ADRC)-Enriched Fat Grafting for Partial Mastectomy Defects: The RESTORE-2 Trial
This prospective trial demonstrates the safety and efficacy of the treatment of breast conservation therapy defects utilizing ADRC-enriched fat grafts. [Eur J Surg Oncol] Abstract | Press Release

Learn More - Reduce Costs Associated With Collecting And Culturing Cells By Using Frozen Primary Cells


Inovio Pharmaceuticals’ PENNVAX®-B HIV Vaccine Demonstrates Strong T-Cell Immune Responses in Therapeutic Vaccine Trial
Inovio Pharmaceuticals, Inc. announced that it has achieved strong T cell immune responses in a Phase I clinical study of PENNVAX®-B, its product for the treatment of the HIV subtype prevalent in North America and Europe, in HIV-positive subjects. [Press release from Inovio Pharmaceuticals, Inc. discussing research presented at the Vaccine World Summit 2012, Hyderabad] Press Release

Alnylam Initiates Phase I Clinical Study of ALN-TTR02, an RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis
Alnylam Pharmaceuticals, Inc. announced that it has initiated dosing in its Phase I clinical trial with ALN-TTR02. [Alnylam Pharmaceuticals, Inc.] Press Release

Tech Bulletin: Expansion Of Hematopoietic Stem And Progenitor Cells


111 Organizations Call for Synthetic Biology Moratorium
Synthetic biology needs more oversight, and the government needs to put in place regulations specific for this field. That is the bottom line for 111 environmental, watchdog, and other organizations that released a report with specific recommendations for managing new biological techniques for building and remaking organisms for research and commercial uses ranging from medicines to biofuels. [ETC Group, Canada] Press Release

Putin Promises Science Boost
Vladimir Putin’s government will aim to establish several “world-class” research universities by 2020. Putin also promised a substantial increase in public funding of basic and applied research. [Russian President, Russia] Press Release

China’s Budget Backs Science
The Chinese government’s promise that researchers will enjoy another year of increased funding was not unexpected, given the country’s relatively buoyant economy and high regard for science. But some scientists fear that too little money will be spent on good-quality basic research, and that too much will be wasted in a funding system in which good connections often trump academic expertise. [National People’s Congress, China] Press Release

Scientists Decry Spanish Cutbacks
Scientists are warning Spain’s government that its planned budget cuts and hiring freeze could be fatal for the nation’s science. [Investigación Digna, Spain] Press Release

U.K. Groups Grapple with Ethical Issues Linked to Clinical Sequencing
The Wellcome Trust Sanger Institute and research partners have a two-prong survey under way involving healthcare professionals and patients. The five-year Genome Ethics survey is designed to gather empirical data that helps guide policy decision-making on whole-genome research studies. [Wellcome Trust Sanger Institute, United Kingdom] Press Release

No Ticket to Ride: U.K. Animal Rights Campaigners End Ferry Service for Lab Animals
Following a high-pressure campaign from animal rights activists, the final ferry company that carried research animals between the United Kingdom and Europe has stopped the service, academic funders announced. [National Anti-Vivisection Alliance, United Kingdom] Press Release

Phacilitate Immunotherapy Leaders' Forum 2012


Merck Partners with Academic Scientists and Biotechnology Entrepreneurs to Create the California Institute for Biomedical Research (Calibr)
Merck announced a collaboration to create Calibr, an independent, not-for-profit organization established to accelerate the translation of basic biomedical research into innovative, new medicines to treat disease. [Merck & Co., Inc.] Press Release

Columbia and Weill Cornell Join National Effort to Spur Clinical Trials in the Neurosciences
The National Institute for Neurological Disorders and Stroke has selected Columbia University College of Physicians and Surgeons and Weill Cornell Medical College to participate in a new national effort to accelerate the development of therapies for people with neurological diseases. [Columbia University Medical Center]
Press Release

Marina Biotech and ProNAi Therapeutics Announce License Agreement for the Development of DNAi-Based Therapeutics
Marina Biotech, Inc. and ProNAi Therapeutics, Inc. announced that the Companies have entered into an Exclusive License Agreement regarding the development and commercialization of DNA interference (DNAi)-based therapeutics utilizing Marina Biotech’s novel SMARTICLES(R) liposomal delivery technology. [Marina Biotech, Inc.] Press Release

DSM and Agennix Sign New Contract for Commercial Manufacture of Talactoferrin
DSM Pharmaceutical Products (DSM) and Agennix AG have signed a new contract under which DSM will continue to manufacture talactoferrin for Agennix at commercial levels. [DSM Pharmaceutical Products]
Press Release

Orgenesis Announces Agreement to Acquire Autologous Insulin Producing Cell Regeneration Technology
Orgenesis Inc. announced that pursuant to a licensing agreement with Tel Hashomer – Medical Research, Infrastructure and Services Ltd. (THM), the Company has an exclusive license to develop and commercialize THM’s rights in functional autologous insulin producing cells regeneration technology. [Orgenesis Inc.]
Press Release

Ottawa Researchers to Lead World-First Clinical Trial of Stem Cell Therapy for Septic Shock
A team of researchers from the Ottawa Hospital Research Institute and the University of Ottawa has been awarded $367,000 from the Canadian Institutes of Health Research and $75,000 from the Stem Cell Network to lead the first clinical trial in the world of a stem cell therapy for septic shock. [Ottawa Hospital Research Institute] Press Release

Prima Receives TGA Manufacturing License for CVac™ in Australia
Prima BioMed Ltd announced that it has received a manufacturing license from the Therapeutic Goods Administration (TGA) to produce the CVac™ immunotherapy ovarian cancer vaccine in Australia. [Prima BioMed Ltd] Press Release

Alnylam Retains Key Claims for Kinesin Spindle Protein (KSP) RNAi Patent in Interference Proceedings
Alnylam Pharmaceuticals, Inc. announced that the United States Patent and Trademark Office has maintained key claims in the company’s KSP RNAi patent in interference proceedings. [Alnylam Pharmaceuticals, Inc.] Press Release

Largest Gene Therapy Trial for Cystic Fibrosis Begins
British scientists are to carry out the largest trial anywhere in the world of a gene therapy for cystic fibrosis. A consortium of researchers from Oxford University, Imperial College London and the University of Edinburgh will start enrolling patients on the trial this month. [University of Oxford] Press Release

Washington Center for Pain Management Begins Enrollment in United States Stem Cell Therapy Study in Subjects with Chronic Back Pain
Washington Center for Pain Management is participating in a nationwide Food and Drug Administration-cleared adult stem cell study testing novel treatment for chronic low back pain and has enrolled its first patient. [PR Newswire] Press Release

Oncothyreon Initiates Phase I Trial of Therapeutic Vaccine Product Candidate ONT-10
Oncothyreon Inc. announced that the first patient has been enrolled in a Phase I trial of ONT-10, a therapeutic vaccine directed at MUC1. [Oncothyreon Inc.] Press Release

BioCardia and Juventas Announce Phase II Development Program of JVS-100 Delivered with the Helical Infusion System to Treat Heart Failure
BioCardia and Juventas Therapeutics announced that they will continue to work together to execute Juventas’ Phase II trial of JVS-100 for the treatment of heart failure. [BioCardia, Inc.] Press Release

Kristi Kiick Selected as AIMBE Fellow
Kristi Kiick, deputy dean of engineering and professor of materials science and engineering and biomedical engineering at the University of Delaware, has been inducted into the American Institute for Medical and Biological Engineering (AIMBE) College of Fellows, Class of 2012. [University of Delaware] Press Release

A*STAR Researchers Amongst Top Young Innovators in Asia-Pacific
A*STAR scholars Dr Jonathan Loh Yuin-Han and Dr Joel Yang have won the inaugural [email protected] Award that recognizes young innovators in the Asia-Pacific region, under the age of 35 years. [Agency for Science, Technology and Research (A*STAR)] Press Release

World Gene Therapy Congress Europe 2012


Notice of Change in Participation of NIH Institutes and Centers in PAR-10-225 “Biomedical Technology Research Center (P41)” (NOT-OD-12-072)

NINDS Policy for Submission of Applications Containing Clinical Trials (NOT-NS-12-007)

The NCI Transition Career Development Award (K22) (PAR-12-121)

National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2012-6345)

February 28-29, 2012: Vaccines and Related Biological Products Advisory Committee Meeting: Announcement: Summary Minutes


Food and Drug Administration (United States)

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Cooperative Manufacturing Arrangements for Licensed Biologics (FR Doc No: 2012-6021)

Revision of Organization and Conforming Changes to Regulations (FR Doc No: 2012-6517)

Medicines and Healthcare Products Regulatory Agency (United Kingdom)

MHRA Publishes Fifth Report on Better Regulation of Medicines Initiative

Therapeutic Goods Administration (Australia)

Advisory Committee on Biologicals (ACB)


NEW The Jackson Laboratory: Making Sense of Mouse Nomenclature and Promoting Genetic Stability
March 29, 1:00PM (ET in US)

NEW World Gene Therapy Congress Europe 2012
May 22-23, 2012
London, United Kingdom

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.


Scientist – Pluripotent Stem Cells (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Antibody and Hybridoma Technology Platforms (STEMCELL Technologies, Inc.)

Business Analyst – Product Management (STEMSOFT Software, Inc.)

Senior Development Associate (Genzyme Corporation)

Quality Assurance Manager, GMP (Cellular Dynamics International)

Research Technologist III (Medical College of Wisconsin)

Postdoctoral Position: Manager/Chair in the Beta- Cell Biology and Therapy Unit (Kocaeli University)

Postdoctoral Position in Stem Cell Biology/Neuroscience/Cancer (The Jackson Laboratory)

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