Exosome-based miR-25 or miR-181a delivery reduces ATXN3 aggregation and cytotoxicity in SCA3 mice
July 14, 2023
Spinocerebellar ataxia type 3 (SCA3) is the most common autosomal dominant hereditary ataxia worldwide, with no cure available. Intranuclear aggregation of the mutant expanded ATXN3 protein is a neuropathological hallmark of SCA3 linked to neurotoxicity.