An international team of scientists has demonstrated that a cocktail of drugs that helps “patch” the genetic mutations that cause Duchenne muscular dystrophy (DMD) can reduce the symptoms of a DMD-like disorder in dogs.
A variety of frameshift or nonsense mutations in the DMD gene interfere with the production of the protein dystrophin and cause progressive muscle weakness in patients with DMD. Previous studies have demonstrated that an intravenous exon-skipping drug, which covers up such mutations, could help rescue dystrophin production in mice with a DMD-like condition; intramuscular injections also have been shown to locally restore dystrophin production in 4 patients.
Kuehn BM. Muscular Dystrophy Patch. JAMA. 2009;301(17):1758. doi:10.1001/jama.2009.569