Functional repair of a mutant chloride channel using a trans-splicing ribozyme.

@article{Rogers2002FunctionalRO,
  title={Functional repair of a mutant chloride channel using a trans-splicing ribozyme.},
  author={Christopher S. Rogers and Carlos G Vanoye and Bruce A. Sullenger and Alfred L. George},
  journal={The Journal of clinical investigation},
  year={2002},
  volume={110 12},
  pages={1783-9}
}
RNA repair has been proposed as a novel gene-based therapeutic strategy. Modified Tetrahymena group I intron ribozymes have been used to mediate trans-splicing of therapeutically relevant RNA transcripts, but the efficiency of the ribozyme-mediated RNA repair process has not been determined precisely and subsequent restoration of protein function has been demonstrated only by indirect means. We engineered a ribozyme that targets the mRNA of a mutant canine skeletal muscle chloride channel (cClC… CONTINUE READING
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