Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

@article{AartsmaRus2003TherapeuticAE,
  title={Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.},
  author={Annemieke Aartsma-Rus and Anneke A. M. Janson and Wendy E. Kaman and Mattie Bremmer-Bout and Johan T den Dunnen and Frank Baas and Gert-Jan van Ommen and Judith C. T. van Deutekom},
  journal={Human molecular genetics},
  year={2003},
  volume={12 8},
  pages={907-14}
}
The dystrophin deficiency leading to the severely progressing muscle degeneration in Duchenne muscular dystrophy (DMD) patients is caused by frame-shifting mutations in the DMD gene. We are developing a reading frame correction therapy aimed at the antisense-induced skipping of targeted exons from the pre-mRNA. Despite introducing a (larger) deletion, an in-frame transcript is generated that allows the synthesis of a slightly shorter, but largely functional dystrophin as found in the mostly… CONTINUE READING

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