| Vol. 22.00 – 4 January, 2021 |
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| Scientists showed that the lentiviral codelivery of Streptococcus pyogenes Cas9 mRNA and expression cassettes that encoded a guide RNA that targeted vascular endothelial growth factor A (Vegfa) was efficacious in a mouse model of wet age-related macular degeneration induced by Vegfa. [Nature Biomedical Engineering] |
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| PUBLICATIONSRanked by the impact factor of the journal |
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| The knockout of Usp47 significantly inhibited BCR-ABL and BCR-ABLT315I-induced chronic myelogenous leukemia (CML) in mice with the reduction of Lin–Sca1+c-Kit+ CML stem/progenitor cells. [Nature Communications] |
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| Researchers used chronic lymphocytic leukemia as a model to investigate the interactions between the tumor microenvironment and chimeric antigen receptor T cells. [Molecular Therapy] |
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| The authors systematically inactivated deubiquitinating enzymes (DUBs) in mouse hematopoietic progenitors using in vivo small hairpin RNA screens. They found that multiple DUBs may be individually required for hematopoiesis and identified ubiquitin-specific protease 15 (USP15) as essential for hematopoietic stem cell maintenance in vitro and in transplantations and Usp15 knockout mice in vivo. [Cell Reports] |
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| Investigators report a potential nanomedicine-based gene therapy for idiopathic pulmonary fibrosis based on regulated macrophage polarization. [Theranostics] |
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| Researchers used AAV9‐PHP.B, a recently developed synthetic adeno‐associated virus, to deliver the coding sequence of Syne4 into the inner ears of neonatal Syne4−/− mice. [EMBO Molecular Medicine] |
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| MRL/lpr mice were injected with miR-199a-5p agomir to evaluate the effects of miR-199a-5p on splenic CD4+ T cell senescence and disease in vivo. [Theranostics] |
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| Incorporation of layered double hydroxide–miRNA nanoparticles into collagen-nanohydroxyapatite scaffolds resulted in successful overexpression of miRNA in mesenchymal stromal cells, demonstrating the development of an efficacious miRNA delivery platform for gene therapy applications in regenerative medicine. [Pharmaceutics] |
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| Researchers developed a combinatorial system using stable lentiviral delivery of single guide RNA followed by transient transfection of Cas9 mRNA by electroporation in human cord blood-derived CD34+ hematopoietic stem and progenitor cells. [Scientific Reports] |
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| Scientists focus on the concepts of regeneration, hurdles faced in the path of regenerative therapy, neurodegenerative diseases and the idea of using peptides, cytokines, tissue engineering, genetic engineering, advanced stem cell therapy, and polyphenolic phytochemicals to cure damaged tissues and neurodegenerative diseases. [Critical Reviews in Food Science and Nutrition] |
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| F-star Therapeutics, Inc. announced that the first patient has been dosed in its Phase I trial evaluating FS222, a potentially best-in-class bispecific antibody targeting CD137 and PD-L1. [F-star Therapeutics, Inc. (Globe Newswire, Inc.)] |
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| January 7 – 8, 2021 Virtual |
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| Karolinska Institutet – Huddinge, Sweden |
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| H. Lee Moffitt Cancer Center & Research Institute – Tampa, Florida, United States |
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| Axovia Therapeutics – London, England, United Kingdom |
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| Lieber Institute for Brain Development – Baltimore, Maryland, United States |
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| National Institute of Arthritis and Musculoskeletal and Skin Diseases – Washington, DC, United States |
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