Targeting the DNA and RNA That Causes Disease Using Small Molecules and Polymers

Professor Steven C. Zimmerman (Univ. of Illinois, Urbana-Champaign, Dept. of Chemistry)
Tuesday, 31 January 2017 - 11:40am
French Family Science Center 2237

This talk will focus on the design, synthesis, and study of ligands that target DNA and RNA repeat sequences that cause disease. The design aspect involves a rational approach using simple principles of supramolecular chemistry.  Regarding the targets, most of the discussion will be on the DNA and RNA sequences that cause myotonic dystrophy type 1 (DM1), these involving repeating sequences containing mismatched base-pairs. The application of the general strategy will also be discussed in the broader context of other potential targets. Considerable focus will also be on: (1) multi-target drug design, i.e., drugs designed to impact more than one part of the disease pathobiology, (2) polyvalent drug design for increasing affinity and selectivity of the therapeutic agents and (3) strategies for delivering the active agents to cells either through in situ assembly or delivery vehicles.  Strategies for assembling active agents within cells that are being pursued include template-assisted synthesis and the nanoparticle-catalyzed construction of complex structures.