Ontology highlight
ABSTRACT:
SUBMITTER: Galant NJ
PROVIDER: S-EPMC4848561 | biostudies-literature | 2016 Apr
REPOSITORIES: biostudies-literature
Galant Natalie J NJ Bugyei-Twum Antoinette A Rakhit Rishi R Walsh Patrick P Sharpe Simon S Arslan Pharhad Eli PE Westermark Per P Higaki Jeffrey N JN Torres Ronald R Tapia José J Chakrabartty Avijit A
Scientific reports 20160428
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 concentration is in the micromolar range. By immune-targeting sparsely populated TTR misfolding intermediates (i.e. monomers), we achieved fibril inhibition at substoichiometric concentrations. We developed an ...[more]