Small-Molecule-Mediated Cleavage of RNA in Living Cells

Authors

  • Dr. Lirui Guan,

    1. Department of Chemistry, The Scripps Research Institute, Scripps Florida, 130 Scripps Way, 3A1, Jupiter, FL 33458 (USA)
    Search for more papers by this author
  • Prof. Dr. Matthew D. Disney

    Corresponding author
    1. Department of Chemistry, The Scripps Research Institute, Scripps Florida, 130 Scripps Way, 3A1, Jupiter, FL 33458 (USA)
    • Department of Chemistry, The Scripps Research Institute, Scripps Florida, 130 Scripps Way, 3A1, Jupiter, FL 33458 (USA)
    Search for more papers by this author

  • This work was funded by the National Institutes of Health (R01-GM079235-05) and The Scripps Research Institute. M.D.D. is a Camille & Henry Dreyfus Teacher-Scholar. We acknowledge Dr. Jessica L. Childs-Disney for SPR measurements.

Abstract

original image

Killing the message: An approach to direct the cleavage of RNA targets with small molecules in living cells is described (see scheme). A bifunctional small molecule (purple) that recognizes a specific three nucleotide repeat sequence and cleaves that sequence in response to light was shown to be effective at degrading the myotonic dystrophy type 1 (DM1) extended repeat RNAs, thereby affecting biological functions.

Ancillary