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Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia.
Zhang S, Bastille A, Gordo S, Ramesh N, Vora J, McCarthy E, Zhang X, Frank D, Ko CW, Wu C, Walsh N, Amarwani S, Liao J, Xiong Q, Drouin L, Hebben M, Chiang K, Chau BN. Zhang S, et al. Among authors: bastille a. PLoS One. 2022 Sep 20;17(9):e0274774. doi: 10.1371/journal.pone.0274774. eCollection 2022. PLoS One. 2022. PMID: 36126056 Free PMC article.