Gene Therapy & CRISPR Cas 9 [PPMD’s 2016 Connect Conference]
Gene Therapy isn’t new in the world of Duchenne. In fact, we have had gene therapy presentations at the Connect Conference the past 20 years. However, we are now at a point where gene therapy is closer than ever before and a potential treatment opportunity we see emerging quickly. But what is gene therapy? What is CRISPR Cas 9? How are they similar? Different? What questions do we need think about, to ask, when considering these opportunities? This panel discussion at PPMD’s 2016 Connect Conference brought together many of the researchers, industry partners, and others working in this exciting area.