Novel approaches to therapy of Duchenne Muscular Dystrophy
Novel approaches to therapy of Duchenne Muscular Dystrophy - Prosiect
Trosolwg o'r prosiect
This project's long term aim to develop a treatment for DMD by using exon-skipping, an approach which relies on creating artificial patches of DNA to bypass faults in the genetic sequence of the Dystrophin gene. This therapy does not constitute a cure because it relies on helping the body to circumvent the fault rather than completely correcting it.