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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: faro r. 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.
Protocol for the Characterization of the Cytosine-Adenine-Guanine Tract and Flanking Polymorphisms in Machado-Joseph Disease: Impact on Diagnosis and Development of Gene-Based Therapies.
Lopes SM, Faro R, Lopes MM, Onofre I, Mendonça N, Ribeiro J, Januário C, Nobre RJ, Pereira de Almeida L. Lopes SM, et al. Among authors: faro r. J Mol Diagn. 2020 Jun;22(6):782-793. doi: 10.1016/j.jmoldx.2020.03.003. Epub 2020 Mar 20. J Mol Diagn. 2020. PMID: 32205289 Free article.