Duchenne muscular dystrophy is a severe form of muscular dystrophy for which treatment options are mainly limited to the use of glucocorticoids to prolong ambulation and treatment of cardiomyopathy by drugs. However, newer therapeutic options that are under research have shown significant promise. Some such options are: Gene Replacement which involves using adeno-associated virus (AAV) to replace defective genes. Exon Skipping which involves skipping one or more exons in the pre-messenger RNA to maintain the dystrophin reading frame.
Mutation Suppression which involves strategies to generate full-length dystrophin protein by reading through premature stop codons. Dystrophin Surrogates that involves agents to upregulate genes that can act as alternatives to dystrophin. Cell Therapies which involve strategies to deliver pluripotent stem cells or genetically corrected muscle cells to make…
