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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.
Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Wein N, et al. Among authors: gushchina lv. Mol Ther Methods Clin Dev. 2022 Jul 11;26:279-293. doi: 10.1016/j.omtm.2022.07.005. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 35949298 Free PMC article.
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