Duchenne Muscular Dystrophy (DMD): An updated review of common available therapies

Duchenne muscular dystrophy (DMD) is a lethal progressive pediatric muscle disorder and genetically inheritedas an X-linked disease that caused by mutations in the dystrophin gene. DMD leads to progressive muscleweakness, degeneration, and wasting; finally, follows with the premature demise in affected individual’s due torespiratory and/or cardiac failure typically by age of 30. For decades, scientists tried massively to find aneffective therapy method, but there is no absolute cure currently for patients with DMD, nevertheless, recentadvanced progressions on the treatment of DMD will be hopeful in the future.Several promising gene therapies are currently under investigation. These include gene replacement, exonskipping, suppression of stop codons. More recently, a promising gene editing tool referred to as CRISPR/Cas9offers exciting perspectives for restoring dystrophin expression in patients with DMD. This review intents tobriefly describe these methods and comment on their advances. Since DMD is a genetic disorder, it should betreated by replacing the deficient DMD copy with a functional one. However, there are different types ofmutations in this gene, so such therapeutic approaches are highly mutation specific and thus are personalized.Therefore, DMD has arisen as a model of genetic disorder for understanding and overcoming of the challenges ofdeveloping personalized genetic medicines, consequently, the lessons learned from these approaches will beapplicable to many other disorders. This review provides an update on the recent gene therapies for DMD that aim tocompensate for dystrophin deficiency and the related clinical trials.

Keywords:

Duchenne muscular dystrophy , Gene therapy , Exon skipping , Stop codon , CRISPR/Cas9