308 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. 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. 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. 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. Genetics and Genomics
    2. Neuroscience

    A scalable platform for the development of cell-type-specific viral drivers

    Sinisa Hrvatin et al.
    Highly paralleled functional evaluation of enhancer activity in single cells generates new cell-type-specific tools with broad medical and scientific applications.
    1. Human Biology and Medicine
    2. 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.
    1. Neuroscience

    Targeting light-gated chloride channels to neuronal somatodendritic domain reduces their excitatory effect in the axon

    Jessica E Messier et al.
    A high axonal chloride concentration explains why activation of light-gated chloride channels causes neurotransmitter release, and a novel hybrid somatodendritic targeting motif ameliorates this phenomenon and improves their inhibitory function.

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