Cell Therapy News 19.10 March 26, 2018 | |
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TOP STORYStem Cells Treat Macular Degeneration Investigators describe the safe and effective implantation of a specially engineered patch of retinal pigment epithelium cells derived from stem cells to treat people with sudden severe sight loss from wet age-related macular degeneration. [Press release from University of California Santa Barbara discussing online prepublication in Nature Biotechnology] Press Release | Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Bone Marrow Transplantation Stimulates Neural Repair in Friedreich’s Ataxia Mice The authors report the neuro-reparative effects of myeloablative allogeneic bone marrow transplantation in a humanized murine model of the disease. [Ann Neurol] Abstract Scientists investigated the effects of co-injection of glycosaminoglycan-degrading enzymes, singly or in combination, with AAV2 as a method of increasing retinal transduction. Experiments using healthy mice demonstrated that these enzymes enhance retinal transduction. [Mol Ther Methods Clin Dev] Full Article Researchers examined the role of the complement membrane attack complex (MAC) and the NLRP3 inflammasome in the pathogenesis of experimental autoimmune uveitis in normal and C9-/- mice that are incapable of assembling the MAC. [Mol Ther] Abstract Investigators examined whether enhancing neurogranin (Ng) expression affected cognitive functions in 5XFAD mice, an animal model of Alzheimer’s disease. They found that the Ng reductions and cognitive decline observed in 5XFAD mice were restored in mice that were intrahippocampally injected with an Ng-expressing lentiviral vector. [Exp Mol Med] Full Article Scientists compared the performance of “Standard” and “Specialized” cryoprotectant media on various cryopreservation and reanimation outcomes in patient-derived xenograft (PDX) tumors. Standard and Specialized media were compared between overall and matched PDX tumors. [Lab Invest] Abstract | Press Release In an animal model of chronic ethanol intake and relapse-drinking, both the intravenous and intra-cerebroventricular administration of a single dose of mesenchymal stem cell-spheroids inhibited chronic ethanol intake and relapse-like drinking by 80–90%, displaying significant effects over three–five weeks. [Sci Rep] Full Article Longitudinal in vivo chemical exchange saturation transfer-magnetic resonance imaging (CEST-MRI) performed at days 15 and 45 after direct injection or at 1, 60 and 90 days after systemic injection revealed robust CEST contrast in myocardium that was later confirmed to express lysine rich protein by immunostaining. [Sci Rep] Full Article Regulatable Lentiviral Hematopoietic Stem Cell Gene Therapy in a Mouse Model of Parkinson’s Disease Researchers developed a hematopoietic stem cell transplantation-based macrophage-mediated glial cell line-derived neurotrophic factor (GDNF) therapy platform. They introduced a regulatable lentiviral vector in order to allow the expression of human GDNF to be adjusted or stopped by oral administration of doxycycline. [Stem Cells Dev] Abstract | |
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REVIEWSClinical Use of Lentiviral Vectors The authors discuss several aspects of lentiviral vectors that will be of interest to clinicians, including an overview of lentiviral vector development, the current uses of viral vectors as therapy for primary immunodeficiencies and cancers, large-scale manufacturing of lentiviral vectors and long-term follow-up of patients treated with gene therapy products. [Leukemia] Abstract | Full Article Stem Cell Therapy in Asherman Syndrome and Thin Endometrium: Stem Cell-Based Therapy Scientists show evidence that the transplantation of different stem cells with a diverse source in the endometrial zone have effects on endometrium such as declined the fibrotic area, an elevated number of glands, stimulated angiogenesis, the enhanced thickness of the endometrium, better formed tissue construction, protected gestation, and improved pregnancy rate. [Biomed Pharmacother] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSMustang Bio to Present at CAR-T Congress USA Mustang Bio, Inc. announced that Sadik Kassim, Ph.D., Chief Scientific Officer, will deliver an oral presentation entitled “CAR-T Cell Therapy for Brain Tumors: Logistical Challenges and Opportunities”. [Press release from Mustang Bio, Inc. discussing research presented at the 2018 CAR-T Congress USA, Dedham] Press Release | |
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INDUSTRY NEWSHelix BioPharma Corp. announced the completion of the collaboration agreement with ProMab Biotechnologies, Inc. [Helix BioPharma Corp. (GlobeNewswire, Inc.)] Press Release Cesca Therapeutics Inc. announced that the company’s device subsidiary, ThermoGenesis Corp., has filed a patent with the U.S. Patent and Trademark Office for a method of further simplifying the processes of T-cell activation and transduction within the company’s proprietary CAR-TXpress workflow. [Cesca Therapeutics Inc.] Press Release Mass. Eye and Ear Performs First FDA-Approved Gene Therapy Procedure for Inherited Disease Massachusetts (Mass.) Eye and Ear made medical history by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The occasion marks the beginning of a new era in medicine, as it is the first time any FDA-approved gene therapy has been given to a patient for any inherited disease. [Massachusetts Eye and Ear] Press Release BioLife Solutions, Inc. announced that the USPTO has issued a notice of allowance for a second patent application titled “Biologic Stability, Delivery Logistics and Administration of Time and/or Temperature Sensitive Biologic Based Materials”. [BioLife Solutions, Inc.] Press Release Nohla Therapeutics and WuXi AppTec Advanced Therapies have entered into a commercial agreement for production of Nohla’s NLA101, a universal donor, ex vivo expanded hematopoietic stem and progenitor cell product. [Nohla Therapeutics] Press Release Alnylam Pharmaceuticals, Inc. announced that the European Medicines Agency has granted access to its Priority Medicines (PRIME) scheme for lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of PH1. [Alnylam Pharmaceuticals, Inc.] Press Release Transgene published its financial results for 2017 and provided an update on the progress of its clinical pipeline, preclinical pipeline and its technology platforms. [Transgene SA] Press Release | |
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POLICY NEWSASCO Names CAR T-Cell Immunotherapy Its Clinical Advance of the Year The Society urges sustained federal research investment to accelerate the next generation of cancer treatments. [The ASCO Post] Editorial Duke’s Mishandling of Misconduct Prompts New U.S. Government Grant Oversight The U.S. National Institutes of Health imposed unusual new requirements on researchers based at Duke University in Durham, North Carolina, who receive federal funds. The changes are a response to concerns over how the institution handled recent cases involving research misconduct and grant management. [ScienceInsider] Editorial Massive Cyberhack by Iran Allegedly Stole Research from 320 Universities, Governments, and Companies Nine Iranians working on behalf of the Islamic Revolutionary Guard Corps hacked the computers of 7998 professors at 320 universities around the world over the past five years, an indictment filed by a federal grand jury alleges. [ScienceInsider] Editorial Final 2018 Budget Deal Should Help the National Science Foundation in 2019, Too The 4% increase for the National Science Foundation (NSF) in Alexandria, Virginia, in the 2018 omnibus spending bill hammered out by congressional leaders this week may be modest next to what its peer science agencies received. But it does offer NSF officials more breathing room to fund some major initiatives starting next year. [ScienceInsider] Editorial Final 2018 Budget Bill Eases Biomedical Researchers’ Policy Worries The 2018 omnibus spending bill is cheering biomedical researchers. Not only because of the 8.8% raise it gives the National Institutes of Health—its largest in 15 years—but also because it blocks or drops several proposed policy changes that had concerned the community. [ScienceInsider] Editorial
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EVENTSNEW 21st American Society of Gene & Cell Therapy (ASGCT) Annual Meeting Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Postdoctoral Fellow – CAR T (City of Hope) NEW Postdoctoral Fellow – Precision Therapy of Aggressive Lymphoma (AstraZeneca) NEW Postdoctoral Fellow – Leukemia (Necker Institut Enfants Malades) Postdoctoral Positions – Immunology, Genetics and Precision Medicine (Ludwig Boltzmann Institute) Postdoctoral Research Fellow – Hematopoiesis (Fred Hutchinson Cancer Research Center) Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope) Postdoctoral Fellowship – Functional Genomics of Human Hematopoiesis (Boston Children’s Hospital) Postdoctoral Fellow – Cancer Research (Indiana University School of Medicine) Postdoctoral Associate – Cardiovascular Science (Duke University) 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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