Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides.

  title={Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides.},
  author={Thomas A Rando and M H Disatnik and Lucy Z.-H. Zhou},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  volume={97 10},
Chimeric RNA/DNA oligonucleotides ("chimeraplasts") have been shown to induce single base alterations in genomic DNA both in vitro and in vivo. The mdx mouse strain has a point mutation in the dystrophin gene, the consequence of which is a muscular dystrophy resulting from deficiency of the dystrophin protein in skeletal muscle. To test the feasibility of chimeraplast-mediated gene therapy for muscular dystrophies, we used a chimeraplast (designated "MDX1") designed to correct the point… CONTINUE READING
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Neuromuscular Disorders: Clinical and Molecular Genetics, ed

  • E. Bakker, G. Van Ommen
  • 1998

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