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Duchenne muscular dystrophy (DMD) results when the open reading frame of the X linked dystrophin gene (DMD) is disrupted by deletions, duplications, point mutations or other rearrangements, inhibiting the production of dystrophin. In animal experiments, splice switching oligomers have been used to induce exon skipping and restoration of the open reading frame. Preliminary studies in boys with DMD have shown that 2′ O-methyl-ribooligonucleoside-phosphorothioate and phosphorodiamidate morpholino oligomers can each be effective in restoring dystrophin production. Now, a trial in London and …
Provenance and peer review Commissioned; internally peer reviewed.
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