Creating a Toolkit to Accelerate the Development of Gene Editing Therapies

The advent of genome editing is creating the potential to correct the underpinnings of genetic diseases by rewriting the mutations that cause them. Before these emerging technologies can be put to use to treat and cure genetic diseases broadly, a number of challenges must be overcome. These include such things as validating new gene editing technologies, finding ways to deliver them precisely to the right cells and tissues in the body, and developing means to detect off-target effects. The National Institutes of Health created the Somatic Cell Genome Editing program to address broad challenges of gene editing with the goal of accelerating the development of new therapies for a wide range of condition through the creation of a toolkit that could be made available to biomedical researchers. We spoke to Erik Sontheimer, co-chair of the Somatic Cell Genome Editing Consortium's steering committee, about the program, the challenges it is seeking to address, and it potential to accelerate the development of a new generation of genetic medicines. Thanks to Pfizer, Inc., Bluebird, and Novartis Gene Therapies for their support of this podcast, part of our Platforms of Hope: Advances in Gene Therapy and Gene Editing series.