Discovery enables effective use of gene therapy for muscular dystrophies and other 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.
https://medicalxpress.com/news..../2024-11-discovery-e