Substoichiometric inhibition of transthyretin misfolding by immune-targeting sparsely populated misfolding intermediates: a potential diagnostic and therapeutic for TTR amyloidoses

Wild-type and mutant transthyretin (TTR) can misfold and deposit in the heart, peripheral nerves, and other sites causing amyloid disease. Pharmacological chaperones, Tafamidis® and diflunisal, inhibit TTR misfolding by stabilizing native tetrameric TTR; however, their minimal effective concentratio...

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Bibliographic Details
Main Authors: Galant, Natalie J., Bugyei-Twum, Antoinette, Rakhit, Rishi, Walsh, Patrick, Sharpe, Simon, Arslan, Pharhad Eli, Westermark, Per, Higaki, Jeffrey N., Torres, Ronald, Tapia, José, Chakrabartty, Avijit
Format: Online
Language:English
Published: Nature Publishing Group 2016
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4848561/