Synthesis and characterization of potent bivalent amyloidosis inhibitors that bind prior to transthyretin tetramerization

…, SK Palaninathan, JC Sacchettini…

文献索引：Green, Nora S.; Palaninathan, Satheesh K.; Sacchettini, James C.; Kelly, Jeffery W. Journal of the American Chemical Society, 2003 , vol. 125, # 44 p. 13404 - 13414

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被引用次数: 82

摘要

The misfolding of transthyretin (TTR), including rate-limiting tetramer dissociation and partial monomer denaturation, is sufficient for TTR misassembly into amyloid and other abnormal quaternary structures associated with senile systemic amyloidosis, familial amyloid polyneuropathy, and familial amyloid cardiomyopathy. Monovalent small molecules that bind to one or both of the unoccupied thyroid hormone binding sites at the TTR quaternary ...