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Abstract #4510

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

Sean C. Forbes1, Pratiksha Awale1, Chris Lopez1, Rebecca Willcocks1, Donovan Lott1, Jeff Chamberlain2, Krista Vandenborne1, and Glenn Walter1
1University of Florida, Gainesville, FL, United States, 2University of Washington, Seattle, WA, United States

Synopsis

Keywords: Muscle, Muscle

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.

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Keywords