Turning Genes into Medicine

Scientists are using multiple strategies to alter or replace problem genes causing disease. These gene therapies are among the hottest areas of research 

Advances in technology and years of trial and error have brought us to a point where we finally have regulatory-approved gene therapies—which generally speaking are therapies that mediate their effect through transcription or translation of genetic material, or specifically alter host genetic sequences to treat or cure disease. There are now gene therapies that treat a handful of disorders including neuromuscular diseases and inherited blindness.

This video story describes one of those approaches being used to design a customized therapy for Duchenne muscular dystrophy, a rare neuromuscular disorder. The work is part of a large collaboration of commercial and academic scientists, led by a foundation devoted to finding cures for Duchenne and other diseases like it.