Gene Therapy: A Closer Look (March 2018)

It has been thrilling to see the first Duchenne patients dosed in gene therapy, an approach that with your help PPMD has been supporting over the last years. But along with the excitement comes questions, concerns, and misunderstandings about some of the newer concepts that accompany gene therapy. Dr. Timothy Cripe from Nationwide Children’s Hospital joined PPMD on March 14, 2018 to answer some of the questions that are top of mind for our community right now, as gene therapy trials start. The webinar covered different virus types, understanding the screening process, dosing, dystrophin constructs, promoters, and how the virus and gene that is delivered integrate into the muscle cell.