Gene therapy for Duchenne muscular dystrophy: an update on the latest clinical developments.

Abstract excerpt

INTRODUCTION: Duchenne muscular dystrophy (DMD) is one of the most severe and devastating neuromuscular hereditary diseases with a male newborn incidence of 20 000 cases each year. The disease caused by mutations (exon deletions, nonsense mutations, intra-exonic insertions or deletions, exon duplications, splice site defects, and deep intronic mutations) in the DMD gene, progressively leads to muscle wasting and...

Topics

• Infant, Newborn
• Humans
• Male
• Muscular Dystrophy, Duchenne
• Dystrophin
• Mutation
• Exons
• Genetic Therapy