Targeting nuclear RNA for in vivo correction of myotonic dystrophy

  title={Targeting nuclear RNA for in vivo correction of myotonic dystrophy},
  author={Thurman M. Wheeler and Andrew J. Leger and Sanjay K Pandey and A Robert Macleod and Masayuki Nakamori and Seng H. Cheng and B. M. Wentworth and C. F. Bennett and Charles A. Thornton},
Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-of-function effects. In the hereditary degenerative disease myotonic dystrophy type 1 (DM1), transcripts from the mutant allele contain an expanded CUG repeat and are retained in the nucleus. The mutant RNA exerts a toxic gain-of-function effect, making it an appropriate target for therapeutic ASOs. However, despite improvements in ASO chemistry and design, systemic use of… CONTINUE READING
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