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Viral-based animal models in polyglutamine disorders.
Henriques C, Lopes MM, Silva AC, Lobo DD, Badin RA, Hantraye P, Pereira de Almeida L, Nobre RJ. Henriques C, et al. Brain. 2024 Apr 4;147(4):1166-1189. doi: 10.1093/brain/awae012. Brain. 2024. PMID: 38284949 Review.
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: henriques c. 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
miRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3.
Nobre RJ, Lobo DD, Henriques C, Duarte SP, Lopes SM, Silva AC, Lopes MM, Mariet F, Schwarz LK, Baatje MS, Ferreira V, Vallès A, Pereira de Almeida L, Evers MM, Toonen LJA. Nobre RJ, et al. Among authors: henriques c. Nucleic Acid Ther. 2022 Jun;32(3):194-205. doi: 10.1089/nat.2021.0020. Epub 2021 Dec 7. Nucleic Acid Ther. 2022. PMID: 34878314 Free PMC article.