4,006 results found
    1. Chromosomes and Gene Expression

    Therapeutic effects of telomerase in mice with pulmonary fibrosis induced by damage to the lungs and short telomeres

    Juan Manuel Povedano et al.
    Telomerase gene therapy represents a novel effective treatment for pulmonary fibrosis associated with short telomeres by improving pulmonary function, decreasing inflammation and accelerating fiber disappearance in fibrotic lungs.
    1. Neuroscience

    Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome

    Mirko Luoni et al.
    Global brain transduction of the instability-prone Mecp2 transgene by systemic AAV-PHP.eB administration is safe and effective in protecting male and female Mecp2 mutant mice from the Rett syndrome disease phenotype.
    1. Neuroscience
    2. Stem Cells and Regenerative Medicine

    AAV ablates neurogenesis in the adult murine hippocampus

    Stephen Johnston et al.
    Future work using recombinant AAV as an experimental tool in the dentate gyrus or as a gene therapy for diseases of the central nervous system should be carefully evaluated.
    1. Cancer Biology
    2. Immunology and Inflammation

    Domain-swapped T cell receptors improve the safety of TCR gene therapy

    Michael T Bethune et al.
    Engineered T cell receptors enable targeted immunotherapy with a reduced risk of harmful autoimmunity.
    1. Cancer Biology
    2. Neuroscience

    Estrogen receptor alpha in the brain mediates tamoxifen-induced changes in physiology in mice

    Zhi Zhang et al.
    Estrogen receptor alpha in the hypothalamus is required for the effects of chronic tamoxifen treament on gene expression, thermoregulation, bone, and movement in mice.
    1. Medicine
    2. Neuroscience

    AAV-Txnip prolongs cone survival and vision in mouse models of retinitis pigmentosa

    Yunlu Xue et al.
    AAV-Txnip prolongs vision in mouse models of blindness, enhancing lactate catabolism, mitochondrial health, and ATP production in a condition that likely has a limited glucose supply.
    1. Biochemistry and Chemical Biology

    Rational design of aptazyme riboswitches for efficient control of gene expression in mammalian cells

    Guocai Zhong et al.
    An empirically derived scoring system for identifying optimal communication modules of aptazymes accelerates the development of ligand-responsive RNA genetic switches functional in mammalian cells.
    1. Neuroscience

    SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration

    Yo Sasaki et al.
    A mouse model of retinal degeneration reveals a common mechanism for axonal degeneration and photoreceptor cell death and identifies SARM1 as a therapeutic candidate for retinopathies.
    1. Cell Biology

    Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1

    Ling Luo et al.
    The eye produces a protein that inhibits the growth of blood vessels in the deep retina, which includes the photoreceptor layer, and disruption of this process can lead to blindness.
    1. Microbiology and Infectious Disease
    2. Structural Biology and Molecular Biophysics

    Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR

    Nancy L Meyer et al.
    Cryo-electron microscopy reveals interactions at the interface between adeno-associated virus and its cellular receptor at 2.4Å resolution.

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