{"id":{"repo_id":"uiuc","oai_identifier":"oai:www.ideals.illinois.edu:2142/42196"},"canonical_url":"https://search.dev.ndltd.org/etd/uiuc/oai:www.ideals.illinois.edu:2142/42196","repository":{"repo_id":"uiuc","name":"University of Illinois - Urbana-Champaign","base_url":"https://www.ideals.illinois.edu/oai-pmh"},"display":{"title":"Identification and investigation of RNA-binding ligands","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.","abstract_html":"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.","abstract_has_math":false,"creators":["Richardson, Stacie"],"institution":"University of Illinois at Urbana-Champaign","degree_name":"Ph.D.","degree_level":"Dissertation","degree_discipline":"Chemistry","degree_department":null,"school":null,"contributors":["Baranger, Anne M.","Zimmerman, Steven C.","Katzenellenbogen, John A.","van der Donk, Wilfred A."],"advisors":[],"committee_chairs":[],"committee_members":[],"year":2013,"date_issued":"2013-02-03T19:27:34Z","date_published":"2013-02-03T19:27:34Z","updated_at":"2026-07-22T22:25:33Z","subjects":["RNA","Ligand","MBNL1","myotonic dystrophy","trinucleotide repeats","CUG repeats"],"languages":["en"],"rights":["Copyright 2012 Stacie Richardson"],"rights_urls":[],"identifier_entries":[]},"links":{"outbound_url":"http://hdl.handle.net/2142/42196","outbound_label":"Handle","outbound_source":"dc:identifier"},"metadata_groups":[{"id":"people","label":"People","entries":[{"key":"dc:contributor","label":"Contributor","values":["Baranger, Anne M.","Zimmerman, Steven C.","Katzenellenbogen, John A.","van der Donk, Wilfred A."]},{"key":"dc:creator","label":"Author","values":["Richardson, Stacie"]}]},{"id":"academic_context","label":"Academic Context","entries":[{"key":"dc:date","label":"Dc Date","values":["2013-02-03T19:27:34Z","2012-12"]},{"key":"dc:type","label":"Dc Type","values":["text"]},{"key":"thesis:degree_discipline","label":"Discipline","values":["Chemistry"]},{"key":"thesis:degree_level","label":"Degree Level","values":["Dissertation"]},{"key":"thesis:degree_name","label":"Degree Name","values":["Ph.D."]},{"key":"thesis:institution_name","label":"Thesis Institution Name","values":["University of Illinois at Urbana-Champaign"]}]},{"id":"subjects_keywords","label":"Subjects and Keywords","entries":[{"key":"dc:subject","label":"Dc Subject","values":["RNA","Ligand","MBNL1","myotonic dystrophy","trinucleotide repeats","CUG repeats"]}]},{"id":"language_rights","label":"Language and Rights","entries":[{"key":"dc:language","label":"Dc Language","values":["en"]},{"key":"dc:rights","label":"Dc Rights","values":["Copyright 2012 Stacie Richardson"]}]},{"id":"identifiers","label":"Identifiers","entries":[{"key":"dc:identifier","label":"Identifier","values":["http://hdl.handle.net/2142/42196"]}]},{"id":"additional","label":"Additional Metadata","entries":[{"key":"dc:description","label":"Description","values":["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. 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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. 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