Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts.

@article{Langlois2003HammerheadRD,
  title={Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts.},
  author={Marc-Andr{\'e} Langlois and Nan Sook Lee and John J. Rossi and Jack Puymirat},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2003},
  volume={7 5 Pt 1},
  pages={670-80}
}
Myotonic dystrophy type 1 (DM1) is caused by an unstable CTG expansion in the 3' untranslated region (3'UTR) of the myotonic dystrophy protein kinase gene (DMPK). Transcripts from this altered gene harbor large CUG expansions that are retained in the nucleus of DM1 cells and form foci. It is believed that the formation of these foci is closely linked to DM1 muscle pathogenesis. Here we investigated the possibility of using a nuclear-retained hammerhead ribozyme expressed from a modified… CONTINUE READING

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