Cell Therapy News Volume 24.08 | Mar 13 2023

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    2023-03-13 | CTN 24.08


    Cell Therapy News by STEMCELL Technologies
    Vol. 24.08 – 13 March, 2023
    TOP STORY

    Single-Cell Transcriptomic Atlas-Guided Development of CAR-T Cells for the Treatment of Acute Myeloid Leukemia

    Functional validation of established CAR-T cells shows robust in vitro and in vivo efficacy in cell line- and human-derived acute myeloid leukemia models with minimal off-target toxicity toward relevant healthy human tissues.
    [Nature Biotechnology]

    Full Article
    Image of man completing the on-demand CFU assay course on his laptop.
    PUBLICATIONSRanked by the impact factor of the journal

    CD19 CAR Antigen Engagement Mechanisms and Affinity Tuning

    Scientists used cryo-electron microscopy to determine the protein structure of several CAR binders complexed with the extracellular portion of human CD19, a target antigen found on B cell malignancies.
    [Science Immunology]

    Abstract

    Limitations of Gene Editing Assessments in Human Preimplantation Embryos

    To overcome the limitations of analyzing minute embryo DNA, researchers validated gene editing outcomes in stable embryonic stem cells derived from targeted human embryos.
    [Nature Communications]

    Full ArticlePress Release

    An Innovative Hematopoietic Stem Cell Gene Therapy Approach Benefits CLN1 Disease in the Mouse Model

    Scientists provided evidence of the efficacy and feasibility of a novel approach to treat CLN1 disease and possibly other neurodegenerative conditions, paving the way for its future clinical application.
    [EMBO Molecular Medicine]

    Full ArticleGraphical Abstract

    A Small Noncoding RNA Links Ribosome Recovery and Translation Control to Dedifferentiation during Salamander Limb Regeneration

    Researchers identified a link between miRNA regulation, ribosome biogenesis, and protein synthesis during newt limb regeneration.
    [Developmental Cell]

    Full ArticleGraphical Abstract

    Gonadal Function in Pediatric Fanconi Anemia Patients Treated with Hematopoietic Stem Cell Transplant

    Investigators performed a retrospective analysis of 98 pediatric patients with Fanconi anemia who were transplanted between July 1990 to June 2020 to evaluate the incidence of gonadal dysfunction in female and male patients with Fanconi anemia.
    [Haematologica]

    Abstract

    Impact of Allogeneic Stem Cell Transplantation on Thyroid Function

    This observational, cross-sectional study assessed the prevalence of hypothyroidism in adult allo-stem cell transplantation recipients according to time from transplantation, and to identify risk factors.
    [Journal Of Endocrinological Investigation]

    Abstract

    Development of Engineered CAR T Cells Targeting Tumor-Associated Glycoforms of MUC1 for the Treatment of Intrahepatic Cholangiocarcinoma

    Scientists suggested the CAR T cells could specifically eliminate Tn-MUC1-positive intrahepatic cholangiocarcinoma cells, but not Tn-MUC1-negative intrahepatic cholangiocarcinoma cells, in vitro and in vivo.
    [Journal Of Immunotherapy]

    Full Article

    A Multicenter Prospective Interventional Trial of Therapeutic Angiogenesis Using Bone Marrow-Derived Mononuclear Cell Implantation for Patients With Critical Limb-Threatening Ischemia Caused by Thromboangiitis Obliterans

    Researchers evaluated the safety and efficacy of bone marrow-derived mononuclear cell implantation in critical limb-threatening ischemia patients with thromboangiitis obliterans.
    [Circulation Journal]

    Abstract
    How can evaluating the immuno-oncology potential of compounds in vitro predict clinical outcomes? Watch this webinar by Dr. Alison O'Mahony, Eurofins DiscoverX
    REVIEWS

    Immunotherapy for Neuroblastoma by Hematopoietic Cell Transplantation and Post-Transplant Immunomodulation

    The authors oversee the potential benefit of reinforcement of immunotherapy for neuroblastoma following hematopoietic cell transplantation, focusing primarily on lymphocytes and dendritic cells.
    [Critical Reviews In Oncology/Hematology]

    AbstractGraphical Abstract

    INDUSTRY AND POLICY NEWS

    Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes

    Vertex Pharmaceuticals, Inc. announced that the US FDA had cleared the IND for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes.
    [Vertex Pharmaceuticals, Inc.]

    Press Release

    In Wake of Gene-Edited Baby Scandal, China Sets New Ethics Rules for Human Studies

    The birth of babies with genomes edited by Chinese biophysicist He Jiankui has prompted China and other nations to tighten oversight of such germline editing.
    [Science]

    Press Release
    FEATURED EVENT

    Autophagy and Neurodegeneration: Mechanisms to Therapies

    March 26 – 29, 2023
    Snowbird, Utah, United States

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    JOB OPPORTUNITIES

    Postdoctoral Position – Bioinformatics Safety Assessment of Gene and Cell Therapy

    Leiden University – Leiden, Netherlands

    Postdoctoral Position – Cell Fate Plasticity and Cancer

    The German Cancer Re­search Center – Heidelberg, Germany

    Postdoctoral Position – Viral Vectors for Gene and Cell Therapy

    ETH Zurich – Zurich, Switzerland

    Staff Scientist – CAR T Cell Therapy

    City of Hope – Duarte, California, United States

    Director – Cell Therapy Research Operations

    Rutgers Cancer Institute of New Jersey – New Brunswick, New Jersey, United States

    > See All Jobs

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