Volume 13.25 | Jul 2

Issue 13.25 July 2, 2012
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TOP STORY
Genetic Correction of Huntington’s Disease Phenotypes in Induced Pluripotent Stem Cells
Investigators report that human induced pluripotent stem cells (iPSCs) derived from Huntington’s disease patient fibroblasts can be corrected by the replacement of the expanded CAG repeat with a normal repeat using homologous recombination, and that the correction persists in iPSC differentiation into DARPP-32-positive neurons in vitro and in vivo. [Cell Stem Cell] Abstract | Press Release

Increase reproducibility between experiments using frozen primary cells. Learn more.


PUBLICATIONS (Ranked by impact factor of the journal)

Fast-Degrading Elastomer Enables Rapid Remodeling of a Cell-Free Synthetic Graft into a Neoartery
Researchers designed cell-free biodegradable elastomeric grafts that degrade rapidly to yield neoarteries nearly free of foreign materials three months after interposition grafting in rat abdominal aorta. This design focuses on enabling rapid host remodeling. [Nat Med] Abstract | Press Release

Mesenchymal Stem Cells Restore Cortical Rewiring after Neonatal Ischemia in Mice
Researchers investigated the effect of neonatal hypoxic-ischemic brain damage and mesenchymal stem cell treatment on the structure and contralesional connectivity of motor function-related cerebral areas. [Ann Neurol] Abstract

Maturation of Human Embryonic Stem Cell-Derived Pancreatic Progenitors into Functional Islets Capable of Treating Pre-Existing Diabetes in Mice
Scientists developed a protocol to efficiently differentiate commercially available human embryonic stem cells in vitro into a highly enriched PDX1+ pancreatic progenitor cell population that further develops in vivo to mature pancreatic endocrine cells. [Diabetes] Abstract | Press Release

Adoptive Transfer of Immunomodulatory M2 Macrophages Prevents Type 1 Diabetes in NOD Mice
Scientists suggest that immunosuppressive macrophage therapy represents a novel intervention that stops ongoing autoimmune type 1 diabetes and may have relevance in a clinical setting. [Diabetes] Abstract | Press Release

Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy
Scientists reprogrammed fibroblasts and myoblasts from limb-girdle muscular dystrophy 2D patients to generate human induced pluripotent stem cells (iPSCs) and developed a protocol for the derivation of mesoangioblast-like cells from these iPSCs. Transplantation of mouse iPSC-derived mesoangioblasts into a-sarcoglycan-null immunodeficient mice resulted in functional amelioration of the dystrophic phenotype and restoration of the depleted progenitors. [Sci Transl Med] Abstract

Bone Marrow Cell Transplantation Restores Olfaction in the Degenerated Olfactory Bulb
Bone marrow cells were transplanted into P20 mice that exhibit selective olfactory degeneration in adulthood between P60 and P150. The alleviation of mitral cell loss in transplanted mice was accompanied by functional recovery witnessed by significantly improved olfactory detection and enhanced odor discrimination. [J Neurosci] Abstract

Programming of Regulatory T Cells from Pluripotent Stem Cells and Prevention of Autoimmunity
Investigators generated functional regulatory T (Treg) cells from induced pluripotent stem cells and determined the potential role of such cells for Treg cell-based immunotherapy against autoimmunity in a therapeutic setting. [J Immunol] Abstract

Inhibition of Lnk in Mouse Induced Pluripotent Stem Cells Promotes Hematopoietic Cell Generation
Researchers showed that the inhibition of Lnk in embryonic stem and induced pluripotent stem cells could result in the efficient generation and expansion of hematopoietic cells. [Stem Cells Dev] Abstract

Efficient and Simultaneous Generation of Hematopoietic and Vascular Progenitors from Human Induced Pluripotent Stem Cells
Scientists present comparative differentiation results for simultaneously generating hematopoietic and vascular progenitors from both human embryonic stem cells and fibroblast-human induced pluripotent stem cells. [Cytometry A] Abstract

Adipose Derived Mesenchymal Stem Cell Transplantation via Portal Vein Improves Microcirculation and Ameliorates Liver Fibrosis Induced by CCl4 in Rats
Researchers investigated the therapeutic potential of adipose derived mesenchymal stem cell transplantation in liver fibrosis caused by carbon tetrachloride in rats as well as its underlying mechanism, and explored the appropriate infusion pathway. [J Transl Med] Abstract | Full Article

Learn more about the new smartphone app for human blood cell frequencies



SCIENCE NEWS

Rudimentary Liver Grown In Vitro
Scientists have used induced stem cells to create a liver-like tissue in a dish. [Press release from Nature News discussing research presented at the International Society for Stem Cell Research (ISCCR) 10th Annual Meeting, Yokohama] Press Release

Harvard Bioscience’s “InBreath” Bioreactors Used in World’s First Successful Regenerated Laryngotracheal Transplants
Harvard Bioscience, Inc. announced that its “InBreath” bioreactors were used for the world’s first and second successful laryngotracheal implants, using synthetic laryngotracheal scaffolds seeded with cells taken from the patients’ bone marrow. [Harvard Bioscience, Inc.] Press Release

First Patients Dosed in Ib Phase of Neuralstem’s NSI-189 Trial in Major Depressive Disorder
Neuralstem, Inc. announced that the first patients were dosed in Phase Ib of its ongoing trial to test the safety of NSI-189 in the treatment of major depressive disorder (MDD). NSI-189 is a proprietary new chemical entity that stimulates new neuron growth in the hippocampus, a region of the brain believed to be implicated in MDD as well as other diseases and conditions. [Neuralstem, Inc.] Press Release

Air-Liquid Interface Culture for Respiratory Research: Watch Q&A Video


 
POLICY

UNESCO to Set Up UN Science Advisory Board
The UN secretary-general, Ban Ki-moon, has agreed to set up an international scientific advisory board to provide him with guidance on science-related issues, and enable him to provide advice to UN member states on such issues. [UN Environmental, Scientific and Cultural Organization (UNESCO)] Press Release

A University Professor Becomes Egypt’s President
According to his election promises, Mohammed Morsi plans to gradually increase science research spending to 2.5% of GDP. He will link research institutes to industry and promote the protection of intellectual property as a cornerstone to make research more attractive. [Egyptian President, Egypt] Press Release

Science at Stake in Mexican Election
Mexican scientists have watched with dismay as their country, Latin America’s second-largest economy, has slipped down the research-spending ranks in recent years. Candidates in the presidential election have pledged to change that. [Mexican Government, Mexico] Press Release

Developing More Predictive In Vitro Models 3rd Annual Summit



BUSINESS

HemoGenix® FDA Master File to Measure Blood Stem Cell Potency for Cellular Therapy Products: Advanced Tests for Umbilical Cord Blood Stem Cell Transplantation to Help Reduce Engraftment Failure
HemoGenix® announced that FDA CBER has given HemoGenix® its first Master File Number for an in vitro blood stem cell potency, quality and release assay for cellular therapy products used for stem cell transplantation purposes. [HemoGenix, Inc] Press Release

Sirnaomics Received NIH SBIR Grant for Developing Its Scarless Wound Healing siRNA Therapeutics
Sirnaomics, Inc. announced that the company has been awarded another NIH SBIR (Small Business Innovation Research) grant for developing its small interfering RNA (siRNA) therapeutic product STP705 to improve skin wound healing with minimized scar formation. [Sirnaomics, Inc.] Press Release

RXi Pharmaceuticals Announces Initiation of First Clinical Trial
RXi Pharmaceuticals Corporation announced that it successfully initiated its first ever clinical study with an RNA interference (RNAi) compound, RXI-109, using their proprietary self-delivering RNAi technology. RXI-109 has been shown to effectively silence a key regulator of scarring, connective tissue growth factor, in vitro in cell culture and in vivo in rodent skin models. [RXi Pharmaceuticals Corporation] Press Release

International Stem Cell Corporation Reports Reaching Milestone in Its Cornea Program
International Stem Cell Corporation announced that its Research and Development team has advanced its program to create a functional and transplantable human cornea by developing a new method to derive corneal endothelium-like cells from human pluripotent stem cells. [International Stem Cell Corporation] Press Release

Compugen Announces Validation Results for a Second Potential Immune Checkpoint Target for Cancer Immunotherapy
Compugen Ltd. announced results demonstrating the therapeutic potential of CGEN-15022, a Compugen-discovered B7/CD28-like membrane protein, as an immune checkpoint target for treatment of multiple cancers. [Compugen Ltd.] Press Release

Tocagen to Utilize ClearPoint® Neuro Intervention System for MRI-Guided Delivery of Toca 511 in a Study of Patients with Brain Cancer
MRI Interventions’ ClearPoint® Neuro Intervention System will be utilized at selected sites in Tocagen’s ongoing investigational clinical trial for the delivery of Toca 511 into brain tumors under real-time magnetic resonance imaging (MRI) guidance. Toca 511 is a retroviral replicating vector that is designed to deliver a cytosine deaminase gene selectively to cancer cells. [MRI Interventions, Inc.] Press Release

Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Myotonic Dystrophy
Biogen Idec and Isis Pharmaceuticals, Inc. announced that they have entered into an exclusive, worldwide option and collaboration agreement under which the companies will develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1. [Isis Pharmaceuticals, Inc.] Press Release

Osiris Bolsters Its Stem Cell Intellectual Property Estate
Osiris Therapeutics, Inc. announced the expansion of its intellectual property protection around Prochymal®. The United States Patent and Trademark Office granted Osiris two patents that cover multiple mechanisms of action related to cardiac tissue repair. [Osiris Therapeutics, Inc.] Press Release

Alnylam Expands and Develops Management Team
Alnylam Pharmaceuticals, Inc. announced that it has expanded and developed its management team with new appointments and promotions. [Alnylam Pharmaceuticals, Inc.] Press Release

Jennerex Appoints Dr. Laurent Fischer as President and Chief Executive Officer
Jennerex, Inc. announced that Laurent Fischer, M.D., has been appointed to the positions of president, chief executive officer and board member. [Jennerex, Inc.] Press Release



CBER

2012 Advisory Committee Tentative Meetings

Exceptions and Alternative Procedures Approved Under 21 CFR 640.120



REGULATORY

Food and Drug Administration (United States)

Agreements and Memoranda of Understanding between the Food and Drug Administration and Other Departments, Agencies, and Organizations; Withdrawal (FR Doc No: 2012-15713)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-15591)

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2012-15593)

Guidance for Industry on Lupus Nephritis Caused by Systemic Lupus Erythematosus – Developing Medical Products for Treatment; Withdrawal of Guidance (FR Doc No: 2012-15716)



EVENTS

NEW Phacilitate: 9th Annual North American Cell & Gene Therapy Forum 2013
January 28-30, 2012
Washington, United States

NEW
British Society for Gene and Cell Therapy Conference 2013
April 17-19, 2013
London, United Kingdom

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



JOB OPPORTUNITIES

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

Scientist – Epithelial Cell Research (STEMCELL Technologies, Inc.)

Scientist – hPSC Bioengineering (STEMCELL Technologies, Inc.)

Scientist – Mesenchymal Cell Research (STEMCELL Technologies, Inc.)

Research Associate – Chemistry (STEMCELL Technologies, Inc.)

Research Associate – Particle Chemistry (STEMCELL Technologies, Inc.)

Research Technologist – Contract Assay Services (STEMCELL Technologies, Inc.)

Research Technologist – Media Development (STEMCELL Technologies, Inc.)

iPSC Scientist Position – Laboratory of Translational Cell Biology (The Bassell Lab, Emory University)

Chief Scientific Officer (SynGen Inc.)

Cellular Therapy Lab Specialist (The University of Texas Medical School at Houston)

Postdoctoral Research Position for Minority Candidate (New York Obesity Research Center)

Clinical Coordinator (New York Obesity Nutrition Research Center)


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