Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.

Abstract excerpt

Duchenne muscular dystrophy (DMD) is one of the most common and lethal genetic disorders, with 20,000 children per year born with DMD globally. DMD is caused by mutations in the dystrophin (DMD) gene. Antisense-mediated exon skipping therapy is a promising therapeutic approach that uses short DNA-like molecules called antisense oligonucleotides (AOs) to skip over/splice out the mutated part of the gene to produce...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Dog Diseases
• Share to Dogs
• Share to Dystrophin