Advances in gene therapy for muscular dystrophies [version 1; referees: 2 approved]

Abstract

Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre integrity. So far, many approaches have been tested from the traditional gene addition to newer advanced approaches based on manipulation of the cellular machinery either at the gene transcription, mRNA processing or translation levels. Unfortunately, despite all these efforts, no efficient treatments for DMD are currently available. In this review, we highlight the most advanced therapeutic strategies under investigation as potential DMD treatments.

Keywords

• Animal Genetics
• Dermatologic Pharmacology
• Drug Discovery & Design
• Epidemiology
• Medical Genetics
• Musculoskeletal Pharmacology
• Neuromuscular Diseases
• Pharmacokinetics & Drug Delivery