2026-09-21T09:01:38Z https://www.ideals.illinois.edu/oai-pmh

oai:www.ideals.illinois.edu:2142/42196 2023-07-11 col_2142_5131 col_2142_14789 com_2142_5130 com_2142_14788 com_2142_8903

Baranger, Anne M. Baranger, Anne M. Zimmerman, Steven C. Katzenellenbogen, John A. van der Donk, Wilfred A. Richardson, Stacie 2013-02-03T19:27:34Z 2013-02-03T19:27:34Z 2012-12 2013-02-03T19:27:34Z 2012-12 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. Item withdrawn by Mark Zulauf (zulauf@illinois.edu) on 2012-07-13T20:59:47Z Item was in collections: University of Illinois Theses & Dissertations (ID: 1) No. of bitstreams: 2 Richardson_Stacie.docx: 8613309 bytes, checksum: 24b7998e05ba3ee7cbb93fc048a5fe50 (MD5) Richardson_Stacie.pdf: 2432141 bytes, checksum: fa0d0e7103f7a9914cece1d12ab55267 (MD5) Made available in DSpace on 2013-02-03T19:27:34Z (GMT). No. of bitstreams: 3 Stacie_Richardson.pdf: 2432141 bytes, checksum: fa0d0e7103f7a9914cece1d12ab55267 (MD5) license.txt: 4067 bytes, checksum: df03d1f5d6f437d2fcfdf4b4cc7f7161 (MD5) Richardson_Stacie.docx: 8613309 bytes, checksum: 24b7998e05ba3ee7cbb93fc048a5fe50 (MD5) http://hdl.handle.net/2142/42196 en Copyright 2012 Stacie Richardson RNA Ligand MBNL1 myotonic dystrophy trinucleotide repeats CUG repeats Identification and investigation of RNA-binding ligands text Chemistry 1413 Chemistry 0335 University of Illinois at Urbana-Champaign Dissertation Ph.D. PHD:Chemistry -UIUC 10KS0335PHD