Exon skipping therapy for Duchenne muscular dystrophy.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is caused mostly by internal deletions in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. These deletions abrogate the reading frame and the lack of dystrophin results in progressive muscle deterioration. DMD patients experience progressive loss of ambulation, followed by a need for assisted ventilation, and eventual death in...

Topics

• Animals
• Dystrophin
• Exons
• Humans
• Morpholinos
• Muscular Dystrophy, Duchenne
• Mutation
• Oligonucleotides
• RNA Splicing
• Randomized Controlled Trials as Topic