Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides.

@article{Dunckley1998ModificationOS,
  title={Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides.},
  author={Matthew Dunckley and Muthiah Manoharan and P Villiet and Ian C. Eperon and George Dickson},
  journal={Human molecular genetics},
  year={1998},
  volume={7 7},
  pages={1083-90}
}
Deletions and point mutations in the gene encoding the cytoskeletal protein dystrophin and its isoforms cause either the severe progressive myopathy Duchenne muscular dystrophy (DMD) or the milder Becker muscular dystrophy (BMD), largely depending on whether the reading frame is lost or maintained respectively. Frameshift mutations tend to result in a lack of dystrophin at the sarcolemma, destabilization of the membrane and degeneration of skeletal muscle. The mdx mouse is a valuable animal… CONTINUE READING