Files in this item



application/pdfStacie_Richardson.pdf (2MB)
(no description provided)PDF


Title:Identification and investigation of RNA-binding ligands
Author(s):Richardson, Stacie
Director of Research:Baranger, Anne M.
Doctoral Committee Chair(s):Baranger, Anne M.
Doctoral Committee Member(s):Zimmerman, Steven C.; Katzenellenbogen, John A.; van der Donk, Wilfred A.
Department / Program:Chemistry
Degree Granting Institution:University of Illinois at Urbana-Champaign
myotonic dystrophy
trinucleotide repeats
CUG repeats
Abstract:Myotonic dystrophy type 1 is caused by a toxic CUG RNA repeat expansion in the 3’-UTR of the DMPK gene that sequesters a key splicing regulator, MBNL1, preventing normal modulation of alternative splicing of a variety of genes. Targeting these repeats with small molecules could block the sequestration of MBNL1 and restore normal splicing levels, alleviating the pathogenesis of the disease. To that end, I have screened a library of select compounds, chosen for their potential to act as RNA binding ligands. From the initial screen, I identified several promising lead compounds. I proceeded to perform a structure-activity relationship (SAR) study on one of the lead molecules, NSC657704. Based on the results of the SAR, I designed a set of derivatives, synthesized them, and ascertained their inhibitory activity and RNA binding affinity.
Issue Date:2013-02-03
Rights Information:Copyright 2012 Stacie Richardson
Date Available in IDEALS:2013-02-03
Date Deposited:2012-12

This item appears in the following Collection(s)

Item Statistics