In a collaboration with pharmaceutical company Sanofi-Aventis, University of Florida investigators Dr. Andrew Berglund, Dr. Eric Wang and Dr. Kausiki Datta have been awarded $200,000 by MDF to screen for new drugs to treat DM1 and DM2.
The group will first optimize an assay designed to identify compounds that inhibit the transcription of the repeats in the DM1 and/or DM2 genes, and then will work with Sanofi to conduct a high throughput screen to identify drug candidates. By targeting transcription of the repeats, the group hopes that a variety of potential downstream toxic effects will be corrected, from protein sequestration to improper signaling to protein production through RNA translation.
This work builds on a previous discovery by Dr. Berglund and colleagues that the antibiotic Actinomycin D can block transcription of CUG repeats at nanomolar concentrations.
Siboni RB, Nakamori M, Wagner SD, Struck AJ, Coonrod LA, Harriott SA, Cass DM, Tanner MK, and Berglund JA.
Cell Rep. 2015 December 22.