Molecular BiologybioRxivPreprint
AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3
Carina Henriques, Diana Duarte Lobo, Ana Carolina da Silva…·Rui Jorge Nobre
This study evaluates AAV9-miR-ATXN3 in a non-human primate model of SCA3, showing reduced neuropathology through allele-specific silencing of mutant ATXN3.
DOI 10.1101/2025.10.02.680027