The Chamberlain lab studies mechanisms leading to the muscular dystrophies, the structure & function of dystrophin (mutated in DMD, among the most common inherited diseases), and approaches to therapy. We have performed extensive structure/function studies on dystrophin and on how the dystrophin-glycoprotein complex is assembled in striated muscles, and have pioneered methods for gene delivery to skeletal and cardiac muscles. Our group developed miniaturized dystrophin genes that we named “micro-dystrophin” and were also the first to show that AAV vectors could be used for systemic gene delivery to muscle. Significant amelioration of muscular dystrophy has been achieved in animal models for DMD using systemic AAV delivery of micro-dystrophins, or by delivery of CRISPR/Cas9 components to induce gene editing. Several groups are currently planning or conducting human gene therapy trials using Chamberlain lab micro-dystrophins, including Solid Biosciences, Sarepta and Genethon.