Cell Therapy News 20.33 September 30, 2019 | |
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TOP STORYResearchers developed a combinatorial library of ionizable lipid-like materials to identify mRNA delivery vehicles that facilitate mRNA delivery in vivo and provide potent and specific immune activation. [Nat Biotechnol] Abstract | Press Release | |
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PUBLICATIONS(Ranked by impact factor of the journal)CAR T Cells Targeting BAFF-R Can Overcome CD19 Antigen Loss in B Cell Malignancies B cell activating factor receptor-CAR, but not CD19-CAR, T cells demonstrated antitumor effects against an additional CD19 antigen loss primary patient-derived xenograft in vivo. [Sci Transl Med] Full Article Investigators used published genomic and epigenomic data available through ENCODE to redefine enhancer element boundaries of the β-globin locus control region to construct novel ENCODE core sequences. At low vector copy number, vectors containing the ENCODE core sequences were capable of reversing the sickle phenotype in a mouse model of sickle cell disease. [Mol Ther] Abstract Efficient CRISPR/Cas9-Mediated Gene Knockin in Mouse Hematopoietic Stem and Progenitor Cells The authors describe an efficient CRISPR/Cas9-based system to knock in and repair genes in mouse hematopoietic stem and progenitor cells (HSPCs). They corrected a neomycin-disrupted Rag2 gene. The Rag2-corrected HSPCs restored B and T cell development in vivo. [Cell Rep] Full Article | Graphical Abstract An Anti-CD103 Antibody-Drug Conjugate Prolongs the Survival of Pancreatic Islet Allografts in Mice Targeting CD103 with M290-MC-MMAF induced immunosuppression and prolonged the survival of pancreatic islet allografts in mice. [Cell Death Dis] Full Article Human dental pulp stem cells (hDPSCs) were able to commit towards myogenic lineage in vitro and, four weeks after cell injection, hDPSCs engrafted in the external urethral sphincter whose thickness was almost recovered, committed towards myogenic lineage in vivo, promoted vascularization and an appreciable recovery of the continence. [Cell Prolif] Abstract Compared with the untreated control group, the potential regenerative result and anti-inflammatory effects of human umbilical cord matrix-mesenchymal stem cells were evident at all the tested concentrations in rabbits with induced temporomandibular joint osteoarthritis. [Sci Rep] Full Article Scientists evaluated the therapeutic potential of human umbilical cord perivascular cells engineered to produce insulin growth factor like-I (IGF-I) in experimental liver fibrosis and the role of extracellular vesicles in this effect. [Gene Ther] Abstract Superior Human Hepatocyte Transduction with Adeno-Associated Virus Vector Serotype 7 Researchers evaluated adeno-associated virus (AAV) vector transduction from several serotypes in human liver hepatocytes xenografted into chimeric mice. After systemic administration of AAV vectors encoding a GFP transgene in humanized mice, the liver was harvested for either immunohistochemistry staining or flow cytometry assay for AAV human hepatocyte transduction analysis. [Gene Ther] Abstract Subscribe to one of our other 19 science newsletters such as Mesenchymal Cell News & ESC & iPSC News. | |
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REVIEWST Cell Engineering for Adoptive T Cell Therapy: Safety and Receptor Avidity The authors describe how ‘optimal’ T cell receptor/chimeric antigen receptor affinity can increase the safety/efficacy balance of engineered T cells, and discuss simultaneous or sequential infusion of high and low avidity receptors as further options for efficacious but safe T cell therapy. [Cancer Immunol Immunother] Abstract Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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INDUSTRY NEWSNeuralstem, Inc. announced completion of subject enrollment in its Phase II clinical trial of NSI-566 for the treatment of chronic ischemic stroke. [Neuralstem, Inc.] Press Release Kadimastem Announces Promising Interim Results of Cohort A of its Phase I/IIa Clinical Trial in ALS Kadimastem Ltd. announced promising interim results of cohort A of its Phase I/IIa clinical trial for the treatment of patients with amyotrophic lateral sclerosis (ALS). [Kadimastem Ltd.] Press Release BrainStorm Cell Therapeutics Announces Support for FDA Guidelines on ALS Drug Development BrainStorm Cell Therapeutics Inc. supports the US FDA’s publication on September 24, 2019 of “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry.” This document outlines ALS drug development guidelines and potential opportunities to advance novel ALS therapies through innovative clinical trial design and validated clinical outcome measures. [BrainStorm Cell Therapeutics Inc.] Press Release Capricor Therapeutics to Meet with FDA to Discuss CAP-1002 to Treat Duchenne Muscular Dystrophy Capricor Therapeutics announced that it has been granted a Type B End-of-Phase II meeting with the FDA to discuss prespecified interim analysis of the company’s HOPE 2 clinical trial. [Capricor Therapeutics] Press Release | |
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POLICY NEWSA scientific study published this past spring came with damning implications for Chinese scientist He Jiankui, who created the world’s first gene-edited babies: People with the rare genetic variants that He tried to engineer into embryos, the study asserted, had an increased death rate. On Friday, the paper’s senior author said his study was wrong. [STAT News] Editorial NIH Reveals Its Formula for Tracking Foreign Influences The US National Institutes of Health (NIH) thinks it may have figured out how China’s foreign talents recruitment program is undermining its system for making awards and ensuring ethical behavior by its grantees. [ScienceInsider] Editorial The Potent Effects of Japan’s Stem-Cell Policies A five-year regulatory free-for-all in regenerative medicine has given the industry a boost. But patients might be paying the price. [Nature News] Editorial
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REGULATORYNIHProspective Grant of Exclusive Patent License: Capsid-Free AAV Vectors, Compositions, and Methods for Vector Production and Gene Delivery (FR Doc. No:2019-20992) Notice
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EVENTSNEW Indonesia International Institute for Life Sciences (i3L) Conference 2019 Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Postdoctoral Fellow – Cancer Research (BC Cancer) Research Technologist (STEMCELL Technologies Inc.) Assistant Professor – Regenerative Medicine (Washington University School of Medicine) Research Assistant – Cancer Research (BC Cancer Agency) Postdoctoral Fellow – Cancer Research (Baylor College of Medicine) Postdoctoral Fellow – Cancer Therapy (Purdue University) Postdoctoral Fellowship – Cancer Immunotherapy (McGill University) Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State 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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