Duchenne can be caused by a number of genetic mutations (changes). Nationwide Children’s Hospital is investigating scAAV9.U7.ACCA, an exon skipping gene therapy candidate for patients with a duplication of exon 2 in the DMD gene. AAV functions as a delivery vehicle to introduce a DNA sequence carrying a gene encoding a non-coding RNA—that is, a gene that is not translated into a protein. This RNA binds to sequences around exon 2 of the DMD gene as it is being assembled, excluding one or two copies of exon 2 with the goal of a producing a full-length or near full-length dystrophin protein.
A Phase 1/2 trial is currently active.
This program is sponsored by Audentes Therapeutics and has been partially funded by the Beauhawks Foundation with support from Parent Project Muscular Dystrophy.
Webinar: Understanding Gene Therapy, Part 3 - Galgt2 and Dup2As part of our ongoing series on Understanding Gene Therapy, PPMD hosted a webinar with Dr. Kevin Flanigan, Director of the Center for Gene Therapy at Nationwide Children’s Hospital. Dr. Flanigan helped explain two gene therapy technologies that he and his team at Nationwide are exploring: GALGT2 and Dup2. This webinar is the third in our series on Gene Therapy where we are bring leaders in gene therapy technology to you to discuss their latest projects and provide an update on this incredibly exciting and expanding field.