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 concentrat...

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Pubblicato in:Sci Rep
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4848561/
https://ncbi.nlm.nih.gov/pubmed/27122057
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep25080
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