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, Ana Rita Fernandes, Miguel Monteiro Lopes, Audrey Fayard, Caroline Jan, Sophie Lecourtois, Martine Guillermier, Julien Flament, João Castelhano, Miguel S. Castelo-Branco, Rui Caetano Oliveira, Philippe Hantraye, Padmaja Yalamanchili, , 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.
bioRxiv · Oct 2, 2025 · PreprintMolecular Biology