Promising Small Molecule Study for DM2

Published on Wed, 07/23/2014

A team of researchers at the University of Illinois at Urbana-Champaign recently published the results of a study in which they designed small molecules to combat myotonic dystrophy type 2 (DM2). Dr. Katharine Hagerman, Research Associate at Stanford University Neuromuscular Division and Clinics, provided MDF with the summary below. The study was published in ChemMedChem. 

Previous studies have suggested that the main problem in the cells of people with DM2 is an expansion of a CCTG DNA repeat sequence in the ZNF9 gene. This DNA mutation is transcribed into RNA, where it forms abnormal structures that pull other proteins into clumps and prevent them from performing their normal activities.

In this study, researchers redesigned a small molecule that disrupted the improper interaction of repeat-containing RNA with other proteins, but was highly toxic to cells. Their new molecule still disrupted the desired RNA-protein interaction, but was less toxic and was able to enter cells with greater ease.

Future studies will take the small molecules and test them in fruit flies and mice to see if the molecules will be safe in organisms while continuing to disrupt the RNA-protein interaction associated with DM2. Click here to access the abstract and article.