A Toxic RNA Catalyzes the In Cellulo Synthesis of Its Own Inhibitor

Researchers from Dr. Matthew Disney's lab at the Scripps Research Institute of Florida, including Suzanne Rzuczek, PhD, a 2013 MDF Fund-a-Fellow grant recipient, recently published an article describing a new chemical they designed to inhibit the unhealthy repeat-containing RNA molecule seen in myotonic dystrophy type 2. This project was supported by a postdoctoral fellowship awarded by MDF. The study describes the design of a pair of molecules that seek out the unhealthy repeat RNA and attach to it. When both of the molecules attach near each other on the RNA, they join together and permanently attach to each other, forming a strong inhibitor of the RNA. The authors state that they are "using the cell as a reaction vessel and a disease-causing RNA as a catalyst." By this they mean that only cells that have the large DM2 repeat-containing RNA will create their own chemical to inhibit the negative effects of the DM2 RNA. They were able to show that their chemicals reduced the number of unhealthy RNA clumps found in DM2 cells, and were able to partially reverse the improper processing that normally occurs in those with DM2 as a result of the unhealthy RNA.

Click here to read the full article. You can also view a presentation from the 2014 MDF Annual Conference by Dr. Rzuczek where she discusses this research.

09/25/2014