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Photosensitive small molecule-mediated cleavage of RNA

Photosensitive small molecules that bind and cleave RNA could help treat diseases driven by aberrant RNA transcripts, such as myotonic dystrophy type 1 (DM1). DM1 is caused by expanded r(CUG) repeats in dystrophia myotonica-protein kinase (DMPK) mRNA. An N-hydroxypyridine-2(1H)-thione was developed that specifically bound expanded DMPK mRNA and released hydroxyl radicals that cleaved r(CUG) repeats when photoactivated. In cell culture studies, the compound improved a DM1-associated splicing defect and decreased the level of expanded r(CUG) repeats compared with an r(CUG) repeat-binding control compound. Next steps include developing photoactivated small molecules that can target cancer-associated RNA in tumors.

SciBX 6(3); doi:10.1038/scibx.2013.68
Published online Jan. 24, 2013

Covered by issued and pending patents; available for licensing; undisclosed aspects of technology licensed by SMaRT Therapeutics Inc.

Guan, L. & Disney, M.D. Angew. Chem. Int. Ed.; published online Dec. 20, 2012;
Contact: Matthew D. Disney, Scripps Florida, Jupiter, Fla.