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Dystonia: Are animal models relevant in therapeutics?
Meringolo M, Tassone A, Imbriani P, Ponterio G, Pisani A. Meringolo M, et al. Rev Neurol (Paris). 2018 Nov;174(9):608-614. doi: 10.1016/j.neurol.2018.07.003. Epub 2018 Aug 25. Rev Neurol (Paris). 2018. PMID: 30153948 Review.
Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function.
Cruz L, György B, Cheah PS, Kleinstiver BP, Eimer WA, Garcia SP, Sharma N, Ozelius LJ, Bragg DC, Joung JK, Norberto de Souza O, Macedo Timmers LFS, Breakefield XO. Cruz L, et al. Mol Ther Nucleic Acids. 2020 Sep 4;21:1-12. doi: 10.1016/j.omtn.2020.05.009. Epub 2020 May 15. Mol Ther Nucleic Acids. 2020. PMID: 32502938 Free PMC article.