Functional and structural pathologies in skeletal muscle of a rat model of Duchenne muscular dystrophy.

ABSTRACT:

Background-

Duchenne muscular dystrophy (DMD) is a lethal pediatric degenerative muscle disease for which there is no cure. Robust preclinical models that recapitulate major clinical features of DMD are required to investigate efficacy of potential DMD therapeutics. Rat models of DMD have emerged as promising small animal models to accomplish this; however, there have been no comprehensive studies investigating the functional skeletal muscle decrements associated with the modeling of DMD in rats.

Methods-

CRISPR/Cas9 gene editing was used to generate a dystrophin-deficient Sprague-Dawley muscular dystrophy rat (MDR). Biochemical and immunofluorescent analyses were performed to confirm loss of dystrophin in striated muscles of this rat model. In situ and ex vivo

SUBMITTER: Lee YI 

PROVIDER: S-EPMC12458205 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature