Understanding and Ameliorating the TTR Amyloidoses

This chapter contains sections titled: Overview Introduction to Amyloid Diseases Introduction to Transthyretin Mechanism of TTR Amyloidogenesis Ttr Amyloidogenesis Occurs by a Downhill Polymerization Current Understanding of the Etiology of the TTR Amyloid Diseases Influence of the Cellular Proteostasis Network on TTR Amyloidogenesis Disease-Associated TTR Mutants are Thermodynamically Less Stable than Wild-Type TTR Natural Suppression of a TTR-Associated Amyloid Disease: Strong Support for the Amyloid Hypothesis A Surgical form of Gene Therapy is Currently Utilized to Treat FAP a Surgical form of Gene Therapy is Currently Utilized to Treat FAP Selective Small-Molecule Binding to Tetrameric Ttr Imposes Kinetic Stabilization on the Tetramer: A Chemotherapeutic Strategy for the Ttr Amyloidoses Ttr Kinetic Stabilizers must Bind Selectively to TTR Over the Remainder of the Proteome To be Effective References