Potent kinetic stabilizers that prevent transthyretin-mediated cardiomyocyte proteotoxicity.


A valine-to-isoleucine mutation at position 122 of the serum protein transthyretin (TTR), found in 3 to 4% of African Americans, alters its stability, leading to amyloidogenesis and cardiomyopathy. In addition, 10 to 15% of individuals older than 65 years develop senile systemic amyloidosis and cardiac TTR deposits because of wild-type TTR amyloidogenesis… (More)
DOI: 10.1126/scitranslmed.3002473


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