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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: baatje ms. 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.
Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery.
Pietersz KL, Martier RM, Baatje MS, Liefhebber JM, Brouwers CC, Pouw SM, Fokkert L, Lubelski J, Petry H, Martens GJM, van Deventer SJ, Konstantinova P, Blits B. Pietersz KL, et al. Among authors: baatje ms. Gene Ther. 2021 Aug;28(7-8):435-446. doi: 10.1038/s41434-020-0178-0. Epub 2020 Aug 15. Gene Ther. 2021. PMID: 32801344