| Cell Therapy News 20.31 September 16, 2019 | |
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TOP STORYCRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia Scientists transplanted CRISPR-edited CCR5-ablated hematopoietic stem and progenitor cells into a patient with HIV-1 infection and acute lymphoblastic leukemia. The acute lymphoblastic leukemia was in complete remission with full donor chimerism, and donor cells carrying the ablated CCR5 persisted for more than 19 months without gene editing-related adverse events. [N Engl J Med] Abstract | Editorial |
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PUBLICATIONS(Ranked by impact factor of the journal)Targeting Cardiac Fibrosis with Engineered T Cells Adoptive transfer of T cells that expressed a chimeric antigen receptor against fibroblast activation protein resulted in a significant reduction in cardiac fibrosis and restoration of function after injury in mice. [Nature] Abstract | Press Release C11orf46 binding normalized SEMA6A expression and rescued transcallosal dysconnectivity via repressive chromatin remodeling by the SETDB1 repressor complex. [Nat Commun] Full Article The authors describe a ‘self-inactivating CRISPR’ system consisting of a single guide RNA that deactivated the Streptococcus pyogenes Cas9 nuclease in a doxycycline dependent manner. This enabled defined, temporal control of Cas9 activity in any cell-type and also in vivo. [Mol Ther] Abstract | Graphical Abstract Investigators demonstrated that NeuroD1-mediated in situ astrocyte-to-neuron conversion could regenerate a large number of functional new neurons after ischemic injury. [Mol Ther] Abstract | Press Release | Full Article | Graphical Abstract A Muscle Hybrid Promoter as a Novel Tool for Gene Therapy Combined with AAV2/9, the muscle hybrid promoter provided a high in vivo expression level in the skeletal muscles and heart after both intramuscular and systemic delivery. [Mol Ther Methods Clin Dev] Abstract | Full Article Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID Long-term engraftment of vector-transduced hematopoietic cells was achieved in irradiated Artemis-deficient mice following primary and secondary transplantation. Vector-treated mice displayed T and B lymphopoiesis, polyclonal T cells, had structured lymphoid tissues and produced immunoglobulins. [Mol Ther Methods Clin Dev] Abstract | Full Article Macrophages secreting interleukin-4 (M-IL-4) were established and used to treat acute lung injury through pulmonary macrophage transplantation. The results showed that highly sustained levels of IL-4 and M2 macrophage markers were detected in mice lungs following pulmonary M-IL-4 transplantation. [Cell Death Dis] Full Article The authors showed that bone marrow mononuclear cells (BM-MNCs) separated by an automatic cell isolator, which did not have the ability to remove clots, did not attenuate brain atrophy after stroke. In contrast, manually isolated, clot-free BM-MNC exerted therapeutic effects. [Stroke] Abstract Following syngeneic renal sub-capsule islet transplantation in C57/B6 mice, pulsed focused ultrasound improved the function of transplanted islets with diabetic animals rapidly re-establishing glycemic control. [Sci Rep] Full Article Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. |
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REVIEWSCRISPR Technologies for Stem Cell Engineering and Regenerative Medicine Scientists introduce the principles and recent advances of these CRISPR technologies, their delivery vectors and review their applications in stem cell engineering and regenerative medicine. They focus on in vitro stem cell fate manipulation and in vivo applications such as prevention of retinal and muscular degeneration, neural regeneration, bone regeneration, cartilage tissue engineering, as well as treatment of diseases in blood, skin and liver. [Biotechnol Adv] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. |
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INDUSTRY NEWSBe The Match BioTherapies® and Celyad announced a collaboration to advance the clinical trial of Celyad’s non-gene edited allogeneic CAR-T therapies. [Be The Match BioTherapies] Press Release Gamida Cell Ltd. announced the entry into a research agreement to collect and analyze health outcomes data in patients with hematologic malignancies who receive an allogeneic hematopoietic stem cell transplant from various donor sources. [Gamida Cell Ltd.] Press Release Trucode Launched to Advance Next-Generation Gene Editing Platform Trucode Gene Repair, Inc. announced that it is advancing its next-generation in vivo gene editing technology to correct mutations that cause sickle cell disease, cystic fibrosis, and other devastating genetic disorders. [Trucode Gene Repair, Inc.] Press Release Cellular Biomedicine Group Inc. announced that the company held a meeting with investigators in Shanghai to launch the Phase II clinical trial for AlloJoin®, an “off-the-shelf” allogenic human adipose-derived mesenchymal progenitor cell therapy product targeting knee osteoarthritis. [Cellular Biomedicine Group Inc.] Press Release Momotaro-Gene announced the dosing of the first patient in a Phase II clinical trial combining MTG201, the company’s lead therapeutic candidate, with the PD-1 inhibitor nivolumab in patients with relapsed malignant pleural mesothelioma. [Momotaro-Gene Inc. (GlobeNewswire, Inc.)] Press Release DiscGenics’ US Clinical Study of Cell Therapy for Disc Degeneration Passes Halfway Mark DiscGenics, Inc. announced that the first 30 subjects have been safely treated its first-in-human US clinical study of IDCT, an allogeneic, injectable disc cell therapy for mild to moderate degenerative disc disease. [DiscGenics, Inc.] Press Release Prevail Therapeutics Inc. provided an update on the company’s clinical development of PR001, a potentially disease-modifying, single-dose gene therapy in development for Parkinson’s disease with GBA1 mutations and neuronopathic Gaucher disease. [Prevail Therapeutics Inc.] Press Release | |
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POLICY NEWSScience under Maximum Pressure in Iran From travel restrictions and publishing bans to currency collapse, the restoration of US sanctions has left researchers in Iran reeling. [The Scientist] Editorial EU Research Commissioner Named, but Lacks ‘Research’ in Her Title Mariya Gabriel, a conservative politician from Bulgaria, is slated to become the next European research chief- although her job title, unlike that of her predecessors, doesn’t include the words “research” or “science”. [ScienceInsider] Editorial
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EVENTSNEW 8th Annual International Symposium on Regenerative Rehabilitation Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Research Assistant – Cancer Research (BC Cancer Agency) Research Technologist (STEMCELL Technologies Inc.) Scientific Communications Coordinator (STEMCELL Technologies Inc.) Postdoctoral Fellow – Cancer Research (Baylor College of Medicine) Postdoctoral Fellow – Cancer Therapy (Purdue University) Postdoctoral Researcher – Cell Cycles in Stem Cells (University of California, Santa Cruz) Postdoctoral Fellowship – Cancer Immunotherapy (McGill University) Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State University) Postdoctoral Scholar – Gene and Cell Therapy (Stanford University) Research Scientist – Cellular Electrophysiology (Duke University) Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center) 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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