540 results found
    1. Neuroscience

    Stage-dependent remodeling of projections to motor cortex in ALS mouse model revealed by a new variant retrograde-AAV9

    Barbara Commisso et al.
    The projections from discrete areas to motor cortex increase over disease course in motoneuron disease model with selective spatial and temporal patterns.
    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. Medicine
    2. Neuroscience

    tTARGIT AAVs mediate the sensitive and flexible manipulation of intersectional neuronal populations in mice

    Paul V Sabatini et al.
    tTARGIT AAVs offer a new approach to manipulate intersectional populations that produce selective and robust transgene expression and are easily modified.
    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.
    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. 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. Stem Cells and Regenerative Medicine
    2. Genetics and Genomics

    Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

    Rasmus O Bak et al.
    The CRISPR/Cas9 system can be used with recombinant AAV6 donor delivery to facilitate simultaneous, targeted integration into multiple genetic loci in hematopoietic stem and progrenitor cells.
    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

    Optical dopamine monitoring with dLight1 reveals mesolimbic phenotypes in a mouse model of neurofibromatosis type 1

    J Elliott Robinson et al.
    Heterozygous Nf1 knockout mice exhibit distinct dopaminergic phenotypes in vivo, as revealed by the genetically encoded dopamine sensor dLight1, patch clamp electrophysiology, optogenetics, and novel viral tools for morphological analysis.
    1. Neuroscience

    Rhodopsin targeted transcriptional silencing by DNA-binding

    Salvatore Botta et al.
    Photoreceptor genomic binding of a 20 base-pair-long DNA sequence by a synthetic DNA-binding protein turns off Rhodopsin expression.

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