S3 Ep3: Gene Editing: Fighting DMD from Every Angle
Imagine one day if there was a way to treat or even cure a genetic disease with a single injection? Genome-editing technologies provide the possibility to correct genetic problems at their source by precisely editing the DNA of a living organism. These methods have the potential to transform how a variety of diseases are treated, including a group of genetic disorders characterized by muscle weakness also called muscular dystrophies. Duchenne is the most common and severe form, caused by loss of dystrophin, beneath the sarcolemma. The molecular mechanisms of the disease have been extensively investigated since the discovery of the gene in 1986. Joe talks with Hawken Miller and Debra Miller about their own experience with DMD, the factors that inspired Debra to start CureDuchenne, and their goal to remove any barriers to finding a cure. Joe also talks with Lilly scientists, Ruth Gimeno and Tom Hopkins, and the Chief Scientific Officer of Precision BioSciences Derek Jantz, about the promise of gene editing, how it works, and the collaborative research being done to find a cure for one of the most devastating diseases.