New Discovery Enables Gene Therapy for Muscular Dystrophies, Other Disorders
RNA-based technology facilitates effective use for difficult-to-treat, large-gene diseases. Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The genes that are dysfunctional in muscular dystrophies are often extremely large, and current delivery methods can’t courier such substantial genetic loads into the body.