Pentamidine reverses the splicing defects associated with myotonic dystrophy.

@article{Warf2009PentamidineRT,
  title={Pentamidine reverses the splicing defects associated with myotonic dystrophy.},
  author={M. Bryan Warf and Masayuki Nakamori and Catherine M Matthys and Charles A. Thornton and J Andrew Berglund},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={2009},
  volume={106 44},
  pages={18551-6}
}
Myotonic dystrophy (DM) is a genetic disorder caused by the expression (as RNA) of expanded CTG or CCTG repeats. The alternative splicing factor MBNL1 is sequestered to the expanded RNA repeats, resulting in missplicing of a subset of pre-mRNAs linked to symptoms found in DM patients. Current data suggest that if MBNL1 is released from sequestration, disease symptoms may be alleviated. We identified the small molecules pentamidine and neomycin B as compounds that disrupt MBNL1 binding to CUG… CONTINUE READING
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