Cell Therapy News Volume 22.00 | Jan 4 2021

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    Cell Therapy News 22.00 | Jan 4


    Cell Therapy News by STEMCELL Technologies
    Vol. 22.00 – 4 January, 2021
    TOP STORY

    Lentiviral
    Delivery of Co-Packaged Cas9 mRNA and a Vegfa-Targeting Guide RNA Prevents Wet Age-Related Macular Degeneration in Mice

    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]

    Abstract

    Request your free copy of the 'Production of Chimeric Antigen Receptor T Cells' Wallchart
    PUBLICATIONSRanked by the impact factor of the journal

    Targeting
    USP47 Overcomes Tyrosine Kinase Inhibitor Resistance and Eradicates Leukemia Stem/Progenitor Cells in Chronic Myelogenous Leukemia

    The knockout of Usp47 significantly inhibited BCR-ABL and BCR-ABLT315I-induced chronic myelogenous leukemia (CML) in mice with the reduction of LinSca1+c-Kit+ CML stem/progenitor cells.
    [Nature Communications]

    Full Article

    Leukemic
    Extracellular Vesicles Induce Chimeric Antigen Receptor T Cell Dysfunction in Chronic Lymphocytic Leukemia

    Researchers used chronic lymphocytic leukemia as a model to investigate the interactions between the tumor microenvironment and chimeric antigen receptor T cells.
    [Molecular Therapy]

    Abstract

    USP15
    Deubiquitinase Safeguards Hematopoiesis and Genome Integrity in Hematopoietic Stem Cells and Leukemia Cells

    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]

    Full ArticleGraphical Abstract

    Suppressing
    Sart1 to Modulate Macrophage Polarization by siRNA-Loaded Liposomes: A Promising Therapeutic Strategy for Pulmonary Fibrosis

    Investigators report a potential nanomedicine-based gene therapy for idiopathic pulmonary fibrosis based on regulated macrophage polarization.
    [Theranostics]

    Full Article

    Neonatal AAV Gene Therapy Rescues Hearing in a Mouse Model of SYNE4
    Deafness

    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]

    Full ArticleGraphical Abstract

    Human
    Umbilical Cord-Derived Mesenchymal Stem Cell Therapy Ameliorates Lupus through Increasing CD4+ T Cell Senescence via MiR-199a-5p/Sirt1/p53 Axis

    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]

    Full Article

    Layered
    Double Hydroxide as a Potent Non-Viral Vector for Nucleic Acid Delivery Using Gene-Activated Scaffolds for Tissue Regeneration Applications

    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]

    Full Article

    Combined
    Lentiviral- and RNA-Mediated CRISPR/Cas9 Delivery for Efficient and Traceable Gene Editing in Human Hematopoietic Stem and Progenitor Cells

    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]

    Full Article


    Explore scientific events this January with the Science Events Calendar.
    REVIEWS

    Polyphenols
    and Neurodegenerative Diseases: Focus on Neuronal Regeneration

    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]

    Abstract

    INDUSTRY AND POLICY NEWS

    F-star Therapeutics Announces First Patient Dosed in FS222 Phase I Clinical Trial

    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.)]

    Press Release

    FEATURED EVENT

    ISSCR: Vision 2030 A Forecast for the Field

    January 7 – 8, 2021
    Virtual


    > See All Events

    JOB OPPORTUNITIES


    Postdoctoral Researcher – Single-Cell Immunology

    Karolinska Institutet – Huddinge, Sweden

    Technical Director – Cellular Therapies

    H. Lee Moffitt Cancer Center & Research Institute – Tampa, Florida, United States

    Research Manager – Gene Therapy

    Axovia Therapeutics – London, England, United Kingdom


    Postdoctoral Fellow – Stem Cell Biology

    Lieber Institute for Brain Development – Baltimore, Maryland, United States

    Postdoctoral
    Positions – Gene Regulation in Skeletal Muscle Stem Cells and Embryonic Stem Cells

    National Institute of Arthritis and Musculoskeletal and Skin Diseases – Washington, DC, United States

    > See All Jobs

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