Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.
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
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