GAPDH controls extracellular vesicle biogenesis and enhances the therapeutic potential of EV mediated siRNA delivery to the brain.

Analysis of brain tissues revealed almost 40% silencing of the Htt gene in mouse brain cortex and a highly significant decrease of p62 inclusion bodies in the cortical neurons of the treated animals, an important phenotypic HD marker of disease progression (Fig. 4 e, f ). p62 is an important regulatory protein of selective autophagy, and a reduction in p62 aggregates in HD mouse models has previously been shown to restore HD-associated phenotypes 56 .

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