NIH R01 · 2024
Project Summary Gene therapy for Duchenne muscular dystrophy (DMD) is a promising method to treat this devastating disorder, and 3 companies have recently initiated human clinical trials of AAV/micro-dystrophin infusion. This approach has shown significant potential in pre-clinical studies, and early data from the trials are encouraging. It is hoped that DMD gene therapy will be effective in greatly slowing the development of dystrophy in young boys. Nonetheless, there remain a number of critical issues related to current vector delivery outcomes, several of which will be addressed in this renewal application. Our proposed studies are a natural progression of our previous lines of…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.