Metabolic effects of micro-dystrophin gene therapy on dystrophic skeletal muscle in mice and humans evaluated with 31P-MRS

Motivation: It is unclear whether micro-dystrophin gene therapy will improve the metabolic status of dystrophic muscle. Goal(s): The goal of this study is to understand the effects of micro-dystrophin gene therapy on skeletal muscle energetics in dystrophic muscle. Approach: We used 31P-MRS to evaluate energetics and acid-base status in dystrophic mice and boys with Duchenne muscular dystrophy. Results: We observed improved energetics and acid-base status after micro-dystrophin gene therapy in dystrophic mice and boys with Duchenne muscular dystrophy. Impact: Overall, these findings support that 31P-MRS may be a valuable tool for evaluating treatments in muscular dystrophies.