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Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient

      Duchenne muscular dystrophy (DMD) is a rapid, progressive disease that usually results in death at around the age of 20, while Becker muscular dystrophy (BMD) is a clinically less severe form of the disease that often has only slight debilitating effects. Deletion mutations in the dystrophin gene have been identified in two thirds of DMD/BMD cases and the clinical progression of DMD or BMD patients can be predicted from whether the deletion disrupts or maintains the translational reading frame of the mRNA [
      • Monaco A.P.
      • Bertelson C.J.
      • Liechti-Gallati S.
      • Moser H.
      • Kunkel L.M.
      An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus.
      ]. In spite of the progress of molecular understanding of DMD, any effective treatment has not yet been established.

      Keywords

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