| Vol. 21.33 – 21 September, 2020 |
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| Using adult and neonatal mouse models of Myotonic dystrophy type I, scientists showed that intramuscular or systemic injections of adeno-associated virus vectors encoding nuclease-dead Cas9. [Nature Biomedical Engineering] |
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| PUBLICATIONSRanked by the impact factor of the journal |
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| Scientists investigated the role of perforin in anti-CD19 chimeric antigen receptor T cell efficacy and hemophagocytic lymphohistiocytosis-like toxicities in a syngeneic murine model. [Journal of Clinical Investigation] |
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| Researchers developed a novel formula to improve the expansion of peripheral γδ T cells from healthy donors. They used a humanized mouse model to validate the therapeutic efficacy of expanded γδ T cells in vivo, and the expanded γδ T cells were adoptively transferred into late-stage liver and lung cancer patients. [Cellular & Molecular Immunology] |
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| Scientists used an in vivo implantation model to analyze how immunomodulation via an IL-4 eluting implant affected distinct macrophage populations at the tissue-implant interface and how this may affect downstream regenerative processes. [Biomaterials Science] |
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| Investigators compared outcomes of patients given either allogeneic (allo)-hematopoietic stem cell transplantation (HCT) or autologous (auto)-HCT. Inclusion criteria consisted of adult patients with de novo AML, isolated trisomy 8, first HCT between 2000 and 2018, complete remission at transplantation, and either auto-HCT or allo-HCT with a HLA-identical sibling donor or a 10/10 HLA-matched unrelated donor. [Bone Marrow Transplantation] |
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| Scientists demonstrated that direct delivery of large amounts of transgene encoding guide RNA and repair template DNA via intra-ventricular injection of adeno-associated virus promotes precise targeted genome replacement in adult murine cardiomyocytes expressing Cas9. [Scientific Reports] |
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| Scientists engineered chimeric antigen receptor (CAR) T cell to co-express IL-21 and studied the effects of IL-21 on CAR T cells specific to CD19 and prostate-specific membrane antigens using an in vitro co-culture model and NSG mice transplanted with B-cell tumors. [Cytotherapy] |
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| The authors investigated the pathophysiology of the bone complications associated with mucopolysaccharidosis II and the effect of lentivirus-mediated gene therapy of hematopoietic stem cells on bone lesions of mucopolysaccharidosis type II mouse models in comparison with enzyme replacement therapy. [Molecular Therapy-Methods & Clinical Development] |
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| Investigators suggested that intracerebral administration of serial doses of cytosine deaminase neural stem cells (CD-NSCs) is safe and feasible and identified a recommended dose for Phase II testing of 150 × 106 CD-NSCs. [Cancer Gene Therapy] |
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| Researchers elucidated the neuroprotective effects of a combination of mannitol pretreatment and stem cell transplantation on stroke-induced neural injury. [Journal of Tissue Engineering and Regenerative Medicine] |
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| When neural stem cells (NSCs) were implanted 2mm lateral from the tumor foci, NSCs co-localized with the GBM within seven days. In models of multi-focal disease, NSCs were found to co-localize with multiple tumors, preferentially migrating to tumor foci closest to the site of NSC implantation. [Tissue Engineering] |
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| The authors summarize the progress made towards the development of gene editing technologies to treat primary immunodeficiency diseases and the optimizations that still need to be implemented to turn genome editing into a next-generation lifesaving treatment for rare monogenic life-threatening disorders. [Human Gene Therapy] |
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| Ipilimumab inhibits CTLA-4 and is being investigated for the treatment of glioblastoma, alone or in combination with other treatment modalities, in various preclinical and clinical studies. The results of the most relevant of which are discussed in this review. [Expert Opinion On Investigational Drugs] |
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| REGENXBIO Inc. announced that the first patient has been dosed in the AAVIATE trial, a Phase II trial to evaluate the suprachoroidal delivery of RGX-314 using the SCS Microinjector for the treatment of wet age-related macular degeneration. [REGENXBIO Inc.] |
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| Kiadis Pharma N.V. announced that Kiadis has received $9.5 million in funding from the Advanced Regenerative Manufacturing Institute’s BioFabUSA program, in partnership with the United States Department of Defense, to fund Kiadis’ K-NK-ID101 program. [Kiadis Pharma N.V.] |
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| Nanyang Technological University – Singapore, Singapore |
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| Dana-Farber Cancer Institute – Boston, Massachusetts, United States |
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| Pennsylvania State University – Philadelphia, Pennsylvania, United States |
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| The University of British Columbia – Vancouver, British Columbia, Canada |
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| Terasaki Research Institute – Los Angeles, California, United States |
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