Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an X chromosome-linked lethal muscular disorder with progressing muscle wasting and weakness caused by mutations in the gene encoding a subsarcolemmal protein dystrophin. For a long time, there was no effective cure; however, advances in molecular biology have allowed the development of radical treatment approaches. Among them, exon-skipping therapy using antisense...

Topics

• Animals
• Clinical Trials as Topic
• Drug Evaluation, Preclinical
• Dystrophin
• Exons
• Genetic Therapy
• Humans
• Muscular Dystrophy, Duchenne
• Mutation
• Phenotype
• Targeted Gene Repair
• Treatment Outcome