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...
Main Authors: | , , , , , , , , , , |
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Format: | Online |
Language: | English |
Published: |
Nature Publishing Group
2016
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Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4848561/ |