ANIMAL MODELS OF DUCHENNE MUSCULAR DYSTROPHY

摘要

The present invention provides transgenic, large non-human animal models of Duchenne muscular dystrophy, Becker muscular dystrophy, and DMD-associated dilated cardiomyopathy, as well as methods of using such animal models in the identification and characterization of therapies for Duchenne muscular dystrophy, Becker muscular dystrophy, and DMD-associated dilated cardiomyopathy.

主权项

1. A transgenic animal comprising a genomic mutation in the dystrophin gene (DMD).