Cell Therapy News Volume 21.33 | Sep 21 2020

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    CTN 21.33 | Sep 21 2020


    Cell Therapy News by STEMCELL Technologies
    Vol. 21.33 – 21 September, 2020
    TOP STORY

    The
    Sustained Expression of Cas9 Targeting Toxic RNAs Reverses Disease Phenotypes in Mouse Models of Myotonic Dystrophy Type 1

    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]

    AbstractPress Release

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    PUBLICATIONSRanked by the impact factor of the journal

    Perforin-Deficient
    CAR T Cells Recapitulate Late-Onset Inflammatory Toxicities Observed in Patients

    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]

    Full ArticleGraphical Abstract

    Allogeneic
    Vγ9Vδ2 T-Cell Immunotherapy Exhibits Promising Clinical Safety and Prolongs the Survival of Patients with Late-Stage Lung or Liver Cancer

    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]

    Full Article

    Distinct
    Macrophage Populations and Phenotypes Associated with IL-4 Mediated Immunomodulation at the Host Implant Interface

    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]

    Abstract

    Better
    Leukemia-Free Survival with Allogeneic Than with Autologous HCT in AML Patients with Isolated Trisomy 8: A Study from the ALWP of the EBMT

    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]

    Abstract

    Adeno-Associated
    Virus-Mediated Gene Delivery Promotes S-Phase Entry-Independent Precise Targeted Integration in Cardiomyocytes

    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]

    Full Article

    Inducible
    Secretion of IL-21 Augments Anti-Tumor Activity of piggyBac-Manufactured Chimeric Antigen Receptor T Cells

    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]

    Abstract

    Ex-Vivo
    Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation

    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]

    AbstractGraphical Abstract

    Feasibility
    of Intracerebrally Administering Multiple Doses of Genetically Modified Neural Stem Cells to Locally Produce Chemotherapy in Glioma Patients

    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]

    Abstract

    Mannitol
    Augments the Effects of Systemical Stem Cell Transplantation without Increasing Cell Migration in a Stroke Animal Model

    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]

    Abstract

    Developing
    Bio-Inspired 3D Models of Brain Cancer to Evaluate Tumor-Homing Neural Stem Cell Therapy

    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]

    Abstract

    The StemSpanâ„¢ T Cell Generation Kit is designed to promote differentiation of HSPCs into T cells in stroma- and serum-free culture conditions.
    REVIEWS

    Gene Editing for the Treatment of Primary Immunodeficiency Diseases

    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]

    Abstract

    Ipilimumab: An Investigational Immunotherapy for Glioblastoma

    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]

    Abstract

    INDUSTRY AND POLICY NEWS

    REGENXBIO
    Announces Dosing of First Patient in Phase II AAVIATEâ„¢ Trial of RGX-314 for the Treatment of Wet AMD Using Suprachoroidal Delivery

    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.]

    Press Release

    Kiadis
    Receives $9.5 Million from the Advanced Regenerative Manufacturing Institute’s BioFabUSA Program to Fund Kiadis K-NK Cell COVID-19 Therapy

    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.]

    Press Release

    FEATURED EVENT

    Cell Biology 2020

    September 23
    Virtual


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    JOB OPPORTUNITIES

    Research Fellow – Neural Cell Therapy

    Nanyang Technological University – Singapore, Singapore

    Research Fellow – Multiple Myeloma

    Dana-Farber Cancer Institute – Boston, Massachusetts, United States


    Postdoctoral Scholar – Biomedical Engineering in Regenerative Medicine

    Pennsylvania State University – Philadelphia, Pennsylvania, United States

    PhD
    Student – Topical Gene Therapy

    The University of British Columbia – Vancouver, British Columbia, Canada

    Research
    Positions – Cancer and Immunotherapy

    Terasaki Research Institute – Los Angeles, California, United States

    > See All Jobs

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