Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1

Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1
Lien Nguyen, Long M. Luu, Shaohong Peng,  Julio F. Serrano,  H. Y. Edwin Chan, Steven C. Zimmerman
J Am Chem Soc. 2015 Oct 16

Researchers from Department of Chemistry at the University of Illinois at Urbana-Champaign and the Chinese University of Hong Kong have designed multi-target agents for myotonic dystrophy type 1 (DM1). These agents are small molecule compounds that have three distinct targets in the cell that all eventually lead to inhibition of the RNA sequence known to cause DM1.

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