Investigations of an inducible intact dystrophin gene excision system in cardiac and skeletal muscle in vivo.

ABSTRACT: We sought here to induce the excision of a large intragenic segment within the intact dystrophin gene locus, with the ultimate goal to elucidate dystrophin protein function and stability in striated muscles in vivo. To this end, we implemented an inducible-gene excision methodology using a floxed allele approach, demarcated by dystrophin exons 2-79, in complementation with a cardiac and skeletal muscle directed gene deletion system for spatial-temporal control of dystrophin gene excision in vivo. Main findings of this study include evidence of significant intact dystrophin gene excision, ranging from ~ 25% in heart muscle to ~ 30-35% in skeletal muscles in vivo. Results show that despite evidence of significant dystrophin gene excision, no significant decrease in dystrophin protein content

SUBMITTER: Bez Batti Angulski A 

PROVIDER: S-EPMC7335168 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature