Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.

ABSTRACT: Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in preclinical models of Duchenne muscular dystrophy^1-6, however, the long-term persistence and safety of these genome editing approaches have not been addressed. Here we show that genome editing and dystrophin protein restoration is sustained in the mdx mouse model of Duchenne muscular dystrophy for 1 year after a single intravenous administration of an adeno-associated virus that encodes CRISPR (AAV-CRISPR). We also show that AAV-CRISPR is immunogenic when administered to adult mice⁷; however, humoral and cellular immune responses can be avoided by treating neonatal mice. Additionally, we describe unintend

SUBMITTER: Nelson CE 

PROVIDER: S-EPMC6455975 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature