286 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. 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.
    1. Neuroscience

    Arcuate nucleus and lateral hypothalamic CART neurons in the mouse brain exert opposing effects on energy expenditure

    Aitak Farzi et al.
    CART exerts differential metabolic effects in response to activation of neurons of the Arc, where CART suppresses energy expenditure, while it enforces the reward characteristics of the LHA.
    1. Neuroscience

    Uncovering temporal structure in hippocampal output patterns

    Kourosh Maboudi et al.
    Machine learning models of coordinated hippocampal ensemble activity during sharp wave ripple activity encode structure that mirrors the place cell map expressed during exploration, and enable a new paradigm for analyzing and understanding this offline activity.

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