Gene Therapy for Duchenne (August 2017)

With trials in gene therapy on the horizon, and terms like “cassette”, “construct”, “promoter”, and “transduce” being thrown around, PPMD wants to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy. This Webinar focuses on understanding the different therapies that are being developed that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9, how are they similar and different, and what are the challenges and limitations for each of them.