Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3.
Rufino-Ramos D, Albuquerque PR, Leandro K, Carmona V, Martins IM, Fernandes R, Henriques C, Lobo D, Faro R, Perfeito R, Mendonça LS, Pereira D, Gomes CM, Nobre RJ, Pereira de Almeida L.
Rufino-Ramos D, et al. Among authors: pereira d, pereira de almeida l.
Mol Ther. 2023 May 3;31(5):1275-1292. doi: 10.1016/j.ymthe.2023.04.001. Epub 2023 Apr 6.
Mol Ther. 2023.
PMID: 37025062
Free PMC article.