...dystrophy (DMD), an X-linked recessive disorder affecting approximately 1 in 5000 males. DMD is caused ...difference in DMD, and it’s estimated that exon skipping therapies would be applicable to 80% of DMD patients...feasible case of DMD. If the safety and efficacy of this approach can be optimized, DMD could become one...age. Despite the amount of research conducted on DMD, there is still no good treatment. The dystrophin...be functional, exon skipping is a good option for DMD treatment. Clinical trials have used oligonucleotide...paired zinc finger nucleases to remove exon 51 in DMD patient myoblasts. They observed a 13% removal rate...exons 45-55 in patient myoblasts; when injected into DMD mice, these cells expressed functional dystrophin...