Cell Therapy News Volume 21.21 | Jun 15 2020

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    2020-06-25 | CTN 21.21


    Cell Therapy News by STEMCELL Technologies
    Vol. 21.21 – 15 June, 2020
    TOP STORY

    Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 For Severe Hemophilia B

    Scientists present longitudinal follow up data of subjects who participated in the first trial of a systemically-delivered adeno-associated viral (AAV) vector. Adult males with severe hemophilia B received an AAV2 vector at doses ranging from 8×1010 – 2×1012 vg/kg to target hepatocyte-specific expression of coagulation factor IX; a subset were followed for 12-15 years post vector administration.
    [Molecular Therapy]

    AbstractGraphical Abstract

    Watch 'collaborating to accelerate COVID-19 research'
    PUBLICATIONSRanked by the impact factor of the journal

    Novel Delivery of Cellular Therapy to Reduce Ischemia Reperfusion Injury in Kidney Transplantation

    The authors investigated the potential capability of multipotent adult progenitor cells in kidney normothermic machine perfusion.
    [American Journal of Transplantation]

    Abstract

    Analysis of Aflibercept Expression in Non-Human Primates following Intravitreal Administration of ADVM-022, a Potential Gene Therapy for Neovascular Age-Related Macular Degeneration

    Researchers explored whether ADVM-022, a recombinant adeno-associated virus vector designed to express aflibercept, could induce anti-VEGF protein levels comparable to those observed following a single bolus intravitreal injection of the standard-of-care aflibercept recombinant protein.
    [Molecular Therapy-Methods & Clinical Development]

    Abstract

    Efficient CRISPR/Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome

    Investigators have established a highly efficient method for precise deletion of critical gene sequences in primary human keratinocytes, based on CRISPR/Cas9-mediated gene editing.
    [Molecular Therapy-Methods & Clinical Development]

    AbstractGraphical Abstract

    Neural Stem Cell Delivery via Porous Collagen Scaffolds Promotes Neuronal Differentiation and Locomotion Recovery in Spinal Cord Injury

    Scientists demonstrated that grafts based on porous collagen-based scaffolds, similar to biomaterials utilized clinically in induced regeneration, could deliver and protect embryonic neural stem cells at spinal cord injury (SCI) sites, leading to significant improvement in locomotion recovery in an experimental mouse SCI model.
    [NPJ Regenerative Medicine]

    Full Article

    Lentiviral Gene Therapy Rescues p47phox Chronic Granulomatous Disease and the Ability to Fight Salmonella Infection in Mice

    Investigators tested the ability of a gene therapy approach to control infection by challenging p47phox-null mice with Salmonella Typhimurium, a leading cause of sepsis in chronic granulomatous diseasep atients, and found that mice reconstituted with lentivirus-transduced hematopoietic stem cells had a reduced bacterial load compared with untreated mice.
    [Gene Therapy]

    Full Article

    Phase I Trial of Expanded, Activated Autologous NK Cell Infusions with Trastuzumab in Patients with HER2-Positive Cancers

    The authors infused expanded, activated autologous natural killer cells to potentiate trastuzumab-mediated antibody-dependent cell cytotoxicity in patients with HER2-positive malignancies.
    [Clinical Cancer Research]

    Abstract

    Comparison of the Regenerative Effects of Bone Marrow/Adipose-Derived Stem Cells in the Asherman Model following Local or Systemic Administration

    Scientists compared the effects of systemic or local intrauterine injection of bone marrow or adipose-derived mesenchymal stem cells on the endometrium in a rat model of Asherman’s syndrome.
    [Journal of Assisted Reproduction and Genetics]

    Abstract

    Recovery of Motricity and Micturition after Transplantation of Mesenchymal Stem Cells in Rats Subjected to Spinal Cord Injury

    Investigators evaluated the effect of human adipose-derived stem cell infusion on impaired hindlimb function and urinary continence after spinal cord contusion in rats.
    [Neuroscience Letters]

    Abstract

    Explore Antibodies for Immunology Research
    REVIEWS

    Advances in the Development of Gene Therapy, Noncoding RNA, and Exosome‐Based Treatments for Tendinopathy

    The authors summarize the current understanding and treatments for tendinopathy. They also highlight recent advances in gene therapy, the potential of noncoding RNAs, such as microRNAs, and exosomes, which are nanometer‐sized extracellular vesicles secreted from cells, for the treatment of tendinopathy.
    [Annals of the New York Academy of Sciences]

    Abstract

    INDUSTRY AND POLICY NEWS

    Ovid Therapeutics Announces Strategic Research Collaboration Focused on Accelerating the Development of New Treatments for Rare Neurological Diseases

    Ovid Therapeutics Inc. announced a strategic research collaboration with Columbia University Irving Medical Center researchers to advance genetic based therapies for a range of rare neurological conditions, complementary to Ovid’s current pipeline.
    [Ovid Therapeutics Inc.]

    Press Release

    City of Hope Signs Licensing Agreement with Scopus BioPharma Inc. to Develop Novel, Targeted Immuno-Oncology Gene Therapy

    City of Hope signed an exclusive, worldwide licensing agreement with Scopus BioPharma Inc. Scopus will further develop and plans to commercialize a City of Hope first-in-class, targeted immuno-oncology gene therapy.
    [City of Hope]

    Press Release

    FEATURED EVENT

    International Society for Stem Cell Research (ISSCR) Virtual Annual Meeting 2020

    2020-06-23 – 2020-06-27
    Virtual

    > See All Events

    JOB OPPORTUNITIES

    Postdoctoral Fellow – Immune and Stem Cell Engineering

    Terasaki Institute for Biomedical Innovation – Los Angeles, California, United States

    Faculty Positions – Regenerative Medicine

    Guangzhou Regenerative Medicine and Health Guangdong Laboratory – Guangzhou, China

    Chair – Bioengineering

    University of Washington – Seattle, Washington, United States

    Lab Director – Cancer Immunotherapy

    Vrije Universiteit Brussels – Brussels, Belgium

    Principal Scientist – Human iPSC Gene Engineering

    Empyrean Therapeutics Ltd – Cambridge, United Kingdom

    Postdoctoral Position – RNA Editing and Immunotherapy

    University of Chicago – Chicago, Ilinois, United States

    Postdoctoral Fellow – CRISPR Research

    Boston Children’s Hospital – Boston, Massachusetts, United States

    Postdoctoral Fellow – Cancer Adoptive Cell Immunotherapy

    Wake Forest School of Medicine – Winston Salem, North Carolina, United States

    Postdoctoral Position – CRISPR/Cas Genetic Engineering

    Aarhus University – Aarhus C, Denmark

    Professor Positions – Biomedical Sciences

    The Chinese University of Hong Kong – Hong Kong, China

    > See All Jobs

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