Cell Therapy News 21.20 June 8, 2020 | |
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TOP STORYResearchers used genome editing to engineer a general platform to improve the safety of future human pluripotent stem cell-derived cell transplantation therapies. [Nat Commun] Full Article | |
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PUBLICATIONS(Ranked by impact factor of the journal)Lentivirus-mediated delivery of GFP or factor VIII (FVIII) cassettes driven by the shortened promoters led to GFP expression mainly in endothelial cells in the liver and to long-term FVIII activity without inhibitor formation in hemophilia A mice. [Haematologica] Abstract Intravenous injection of an adeno-associated virus (AAV) vector into two-week-old glycogen storage disease type IIIa mice blocked glycogen accumulation in both cardiac and skeletal muscles, but not in the liver, accompanied by the improvement of muscle functions. [Mol Ther Methods Clin Dev] Abstract | Full Article | Graphical Abstract Investigators determined the effect of intratympanic administration of rodent bone marrow mesenchymal stem cells on the inner ear in an immunocompetent rat model. [J Clin Med] Abstract | Download Full Article Human Beige Adipocytes for Drug Discovery and Cell Therapy in Metabolic Diseases Following transplantation, beige adipocytes (BAs) increased whole-body energy expenditure and oxygen consumption, while reducing body-weight in recipient mice. Scientists also showed the therapeutic utility of BAs in a platform for high-throughput drug screening. [Sci Rep] Full Article Laminin 511 Precoating Promotes the Functional Recovery of Transplanted Corneal Endothelial Cells The authors explored if laminin 511 improved the therapeutic function of intracameral corneal endothelial cells injection for corneal endothelial dysfunction. [Tissue Eng Part A] Abstract Targeting CD79b for Chimeric Antigen Receptor T-Cell Therapy of B-Cell Lymphomas The expression of CD79b in different B-cell lymphomas was determined. Anti-CD79b CAR T-cells expressing one of two different CARs were generated, and a series of in vitro and in vivo experiments were conducted to assess the CAR T-cell function. [Target Oncol] Abstract A rat diabetic foot ulcer (DFU) model was used to simulate the pathophysiology of clinical patients and to investigate the localization of human umbilical cord mesenchymal stem cells (hUC-MSCs) after intravenous transplantation and its role in DFU healing, so as to evaluate the potential of hUC-MSCs in the treatment of DFU. [Acta Biochim Biophys Sin] Full Article Investigators suggested that both melatonin (MT)-BMSCs and BMSCs moved to brain tissues following intravenous transplantation. However, MT-BMSCs had a significant effect on boosting learning, cognition and memory in comparison with BMSCs. [J Chem Neuroanat] Abstract | Graphical Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWSClinical Trials of Stem Cell Treatment for Spinal Cord Injury The authors focus on the clinical trials that have used stem cells for treating spinal cord injury, and highlights the problems that remain to be solved before the widespread clinical use of stem cells can be adopted. [Int J Mol Sci] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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INDUSTRY NEWSSeattle Children’s Research Institute and CSL Behring announced a strategic alliance to develop stem cell gene therapies for primary immunodeficiency diseases. [Seattle Children’s Research Institute] Press Release Alnylam Pharmaceuticals, Inc. announced positive Phase III results from the ILLUMINATE-A study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – in development for the treatment of primary hyperoxaluria type 1. [Alnylam Pharmaceuticals, Inc.] Press Release Axovant Gene Therapies Ltd. announced that it has completed enrollment in the low-dose cohort of the Phase I/II study for Type II GM1 patients evaluating safety, tolerability, and exploratory measures of efficacy at a dose of 1.5×1013 vg/kg delivered intravenously. [Axovant Gene Therapies Ltd.] Press Release Baylx, Inc. Received US FDA Clearance of IND Application for Cord Tissue Mesenchymal Stem Cells Baylx, Inc. announced that the FDA has cleared Baylx’s Investigational New Drug (IND) application for BX-U001, a fresh, non-frozen human umbilical cord tissue mesenchymal stem cells product in patients with rheumatoid arthritis. [Baylx, Inc.] Press Release Sarepta Therapeutics, Inc. announced positive results from a study of SRP-9003, its investigational gene therapy for limb-girdle muscular dystrophy Type 2E. [Sarepta Therapeutics, Inc.] Press Release Elixirgen Therapeutics, Inc. received confirmation from the FDA that its Investigational New Drug (IND) application for its lead candidate, EXG4217, was approved on May 23, 2020. [Elixirgen Therapeutics, Inc.] Press Release | |
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POLICY NEWSAs Pandemic Pounds US Universities, Federal Support Helps Their Labs Stay Afloat This spring, as the coronavirus pandemic shuttered US higher education, many college presidents began to warn of the dire financial impact of a frozen US economy on their institutions. Their mix of revenue sources largely determined how much they might be hurt. [ScienceInsider] Editorial NIH Grapples with Rush to Claim Billions in Pandemic Research Funds For the second time in just over 10 years, the National Institutes of Health (NIH) is scrambling to hand out billions of dollars in emergency research funding and scientists are rushing to get a piece of the action—even as some confusion and concerns abound. [ScienceInsider] Editorial
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EVENTSIn light of COVID-19, many conferences are being cancelled or postponed. As such: We are suspending new event postings in our newsletters and on Twitter. Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Professor Positions – Biomedical Sciences (The Chinese University of Hong Kong) NEW Postdoctoral Researcher – Regenerative Medicine (National University of Ireland Galway) Postdoctoral Position – CRISPR/Cas Genetic Engineering (Aarhus University) Postdoctoral Fellow – Cancer Adoptive Cell Immunotherapy (Wake Forest School of Medicine) Scientist – Cell Therapy (Athersys, Inc.) Postdoctoral Fellow – CRISPR Research (Boston Children’s Hospital) Postdoctoral Position – RNA Editing and Immunotherapy (University of Chicago) Principal Scientist – Human iPSC Gene Engineering (Empyrean Therapeutics Ltd) Lab Director – Cancer Immunotherapy (Vrije Universiteit Brussels) Scientist – Cancer Immunotherapy (Vrije Universiteit Brussels) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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