Exon-Skipping in Duchenne Muscular Dystrophy.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically described in the 19th century, the genetic foundation of DMD was not discovered until more than 100 years later. This genetic understanding opened the door to the development of genetic treatments for DMD. Over the course of the last 30 years, the research that supports this development has moved into the realm of clinical trials...

Topics

• Dystrophin
• Exons
• Genetic Therapy
• Humans
• Muscular Dystrophy, Duchenne
• Mutation
• Oligonucleotides, Antisense