Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 3 of 3 for “"CUG repeats"”.
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Developing potential drugs for the treatment of myotonic dystrophy: from rational design to lead development
… there are the tri- and tetra-nucleotide repeats, CUG and CCUG, in DM1 and DM2, respectively. This key discovery inspired the development of ligands that inhibit MBNL1-RNA complex formation, allowing MBNL1 to resume its biological functions. The background to the DM disease will be given …
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Identification and investigation of RNA-binding ligands
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 …
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Lead compound discovery for myotonic dystrophy
… CTG expansions lead to formation of stable poly(CUG) mRNA which mislocalize splicing factors such as MBNL1 and lead to missplicing in the cell. One therapeutic strategy is to target such mutant mRNA with small molecules to prevent the sequestration of splicing factors which will prevent the …