Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

@article{AartsmaRus2002TargetedES,
  title={Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.},
  author={Annemieke Aartsma-Rus and Mattie Bremmer-Bout and Anneke A. M. Janson and Johan T den Dunnen and Gert-Jan van Ommen and Judith C. T. van Deutekom},
  journal={Neuromuscular disorders : NMD},
  year={2002},
  volume={12 Suppl 1},
  pages={S71-7}
}
Duchenne muscular dystrophy is primarily caused by frame-disrupting mutations in the Duchenne muscular dystrophy gene which abort dystrophin synthesis. We have explored a gene correction therapy aimed at restoration of the reading frame in Duchenne muscular dystrophy patients. Through the binding of antisense oligoribonucleotides to exon-internal sequences in the pre-mRNA, the splicing can be manipulated in such a manner that the targeted exon is skipped and a slightly shorter, but in-frame… CONTINUE READING
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