Duchenne muscular dystrophy
DMD is a degenerative disease affecting one in 2,000 boys, whereby a genetic mutation prevents the body from producing dystrophin, a protein that muscles need to work properly. Without dystrophin, muscle cells become damaged and weaken. Duchenne is an irreversible, progressive disease with no cure.
It is known that even if a small amount of functional protein can be made, the disease is significantly ameliorated. Current scientific efforts are focused on creating drugs that allow for production of a truncated, but functional dystrophin protein. The FDA has approved one such drug but its efficacy remains uncertain. Among several reasons, only low levels of the drug reach affected muscle. DTx technology appears well suited to solve this problem in Duchenne, as well as other conditions where exon skipping can be used to produce a functional protein.