P.312Functional and histological improvements comparing 4 micro-dystrophin constructs in the mdx mouse model of DMD

Duchenne muscular dystrophy (DMD) is the most common severe childhood form of muscular dystrophy. More than 2000 mutations of the DMD gene are responsible for progressive loss of muscle strength, and ultimately respiratory and cardiac failure. Through head-to-head comparison, functional and histological benefit across different micro-dystrophin constructs was evaluated. We designed 4 unique constructs of rAAVrh74 vector, including use of the...