Antisense-mediated modulation of splicing: Therapeutic implications for duchenne muscular dystrophy

  • Aartsma-Rus A
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While disruption of alternative splicing underlies many diseases, modulation of splicing using antisense oligonucleotides (AONs) can have therapeutic implications. The most notable example is Duchenne muscular dystrophy (DMD), where antisense-mediated exon skipping can restore the open reading frame and allow the synthesis of partly functional dystrophin proteins instead of non-functional ones. This approach is currently tested in early phase clinical trials. In this review the development of the exon skipping approach in patient-derived cell cultures, animal models and patients is described and hurdles that have to be overcome to make this personalized medicine type approach widely applicable are discussed.

Author-supplied keywords

  • Antisense oligonucleotides
  • Clinical trials
  • Exon skipping
  • Neuromuscular disorders
  • Splicing
  • Therapy

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  • Annemieke Aartsma-Rus

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