Which Pediatric Conditions Can Be Treated with Exon Skipping?
One of the most notable applications of exon skipping is in the treatment of Duchenne Muscular Dystrophy (DMD), a severe muscle-wasting disease. DMD is caused by mutations in the dystrophin gene, and exon skipping can help restore the production of a truncated, yet functional, dystrophin protein. Other potential applications include certain types of spinal muscular atrophy (SMA) and cystic fibrosis.
