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TOP STORY |
Gene Therapy Using Lentivirus Promising in Three Youngsters Researchers developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency. They took the patients’ own blood stem cells and, in the laboratory, used the lentiviral vector combined with the normal WASP gene to correct the genetic defect in the blood. After a special treatment to eliminate their defective immune system, the patients received their own blood cells that had been altered to contain the normal WASP gene. [Press release from EurekAlert! discussing online prepublication in Science] Press Release | Abstract |
PUBLICATIONS (Ranked by impact factor of the journal) |
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy The authors used a lentiviral vector to transfer a functional arylsulfatase A (ARSA) gene into hematopoietic stem cells (HSCs) from three presymptomatic patients who showed genetic, biochemical, and neurophysiological evidence of late infantile metachromatic leukodystrophy. After reinfusion of the gene-corrected HSCs, the patients showed extensive and stable ARSA gene replacement, which led to high enzyme expression throughout hematopoietic lineages and in cerebrospinal fluid. [Science] Abstract | Press Release TCR-Ligand koff Rate Correlates with the Protective Capacity of Antigen-Specific CD8+ T Cells for Adoptive Transfer Transient B Cell Depletion Combined with Apoptotic Donor Splenocytes Induces Xeno-Specific T and B Cell Tolerance to Islet Xenografts Injectable Laminin-Functionalized Hydrogel for Nucleus Pulposus Regeneration Iron Administration Before Stem Cell Harvest Enables MR Imaging Tracking after Transplantation Transplantation of Islet-Like Cell Clusters Derived from Human Dental Pulp Stem Cells Restores Normoglycemia in Diabetic Mice Wnt/β-Catenin Signaling Triggers Neuron Reprogramming and Regeneration in the Mouse Retina Heart Grafts Tolerized through Third-Party Multipotent Adult Progenitor Cells Can Be Retransplanted to Secondary Hosts with No Immunosuppression |
REVIEWS |
Safety of Intra-Articular Cell-Therapy with Culture-Expanded Stem Cells in Humans: A Systematic Literature Review The authors aimed to systematically review the literature and provide an overview of reported adverse events of intra-articular treatment with culture-expanded stem cells in humans. [Osteoarthritis Cartilage] Abstract Cell Therapy, a Novel Remedy for Dilated Cardiomyopathy? A Systematic Review Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field. |
SCIENCE NEWS |
First In-Depth Study of Knee Cartilage Repair Technique Shows Improvement in Patient Outcomes and Regeneration of Knee Cartilage A natural tissue graft can spur regeneration of cartilage and improve symptoms in patients who have cartilage damage in their knee. De Novo NT is an FDA approved cartilage repair technique. The product is made out of minced cartilage from organ donors under the age of 13. Unlike cartilage in adults, this young tissue has a high proliferative capacity. Cells from the transplanted cartilage escape or migrate into surrounding area tissues, multiply, and form a new cartilage tissue matrix that integrates with the surrounding host tissue. [Press release from Hospital for Special Surgery discussing research presented at the Annual Meeting of the American Orthopaedic Society for Sports Medicine, Chicago] Press Release Alnylam Reports Positive Top-Line Results for ALN-TTRsc, a Subcutaneously Administered RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis |
POLICY |
NSF Is on a Budget Roll According to a final spending plan recently posted by the agency, the National Science Foundation (NSF)’s budget for the current fiscal year ending on 30 September is $149 million lower than in 2012. But that drop of 2.1%, to $6.884 billion, is less than half the 5% decline for the entire civilian government triggered by the sequestration mandated in the 2011 Budget Control Act. [National Science Foundation, United States] Press Release E.U. Commission Beefs Up Research Partnerships with Industry Outcry Over Plans for ‘Japanese NIH’ From our sponsor: Free mouse hematopoietic progenitors wallchart. Request your copy. |
BUSINESS |
NeoStem Advances Autoimmune Platform through UCSF Collaboration NeoStem, Inc. announced that the Company has executed agreements with the University of California, San Francisco (UCSF) and the laboratories of Jeffrey Bluestone, PhD, and Qizhi Tang, PhD, to collaborate on the development of human regulatory T cells for the treatment of type 1 diabetes, steroid resistant asthma, and organ transplant rejection. [NeoStem, Inc.] Press Release uniQure Signs EU Commercialization Agreement with Chiesi Farmaceutici for First Approved Gene Therapy Treatment, and Announces EUR 45 Million (USD 58 Million) in Equity and Collaboration Financing ViaCyte, Inc. Raises $10.6 Million in a Private Financing to Support Clinical Development of its Cell Therapy Product for Insulin Dependent Diabetes MCW Researcher to Study Main Complication after Bone Marrow Transplantation Calimmune Initiates HIV Stem Cell Study at Two California Research Sites Bellicum Pharmaceuticals Initiates Clinical Trial with Novel Controlled Dendritic Cell Immunotherapy in Metastatic Castrate Resistant Prostate Cancer Pluristem’s South Korean Partner Files IND with Korean FDA for PLX Cells SpinalCyte, LLC Receives Japanese Patent for Spinal Disc Tissue Engineering CFDRC Developing Non-Invasive Stem Cell Analyzer Technology American CryoStem Granted IRB Approval for Adipose Derived Stem Cell Processing Protocols |
NIH |
Senate Spending Panel Approves $31 Billion for NIH in 2014, Restoring Sequester Cuts A Senate spending panel approved a bill giving the National Institutes of Health (NIH) $31 billion in 2014, a figure that would restore this year’s painful 5% cut from sequestration and give the agency a small increase over its 2012 budget. Press Release Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-16337) National Cancer Institute; Notice of Closed Meeting (FR Doc No: 2013-16338) |
CBER |
HPC, Cord Blood BLA 125432 |
REGULATORY |
Food and Drug Administration (United States) |
EVENTS |
NEW The Jackson Laboratory: Beyond Leptin – Emerging Mouse Models of Type 2 Diabetes July 18, 1:00PM (ET in US) Webinar NEW Stem Cell Society Singapore (SCSS) Symposium 2013 Visit our events page to see a complete list of events in the cell, gene and immunotherapy community. |
JOB OPPORTUNITIES |
NEW Postdoctoral Position – Stem Cell Therapy and Epigenetics of Retina (University of Pittsburgh, Ophthalmology) Postdoctoral Fellowship – Immunology (Johns Hopkins University School of Medicine) PhD Positions – Tissue Engineering (8 European Research Institutes) Postdoctoral Fellowship – Organic Synthesis and Medicinal Chemistry (Masaryk University) Director of Cell Processing Facility (S L Collins Associates, Inc.) Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine) |
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