Induced Pluripotent Stem Cell Modeling of Multisystemic, Hereditary Transthyretin Amyloidosis

Familial transthyretin amyloidosis (ATTR) is an autosomal-dominant protein-folding disorder caused by over 100 distinct mutations in the transthyretin (TTR) gene. In ATTR, protein secreted from the liver aggregates and forms fibrils in target organs, chiefly the heart and peripheral nervous system,...

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Bibliographic Details
Main Authors: Leung, Amy, Nah, Shirley K., Reid, Whitney, Ebata, Atsushi, Koch, Clarissa M., Monti, Stefano, Genereux, Joseph C., Wiseman, R. Luke, Wolozin, Benjamin, Connors, Lawreen H., Berk, John L., Seldin, David C., Mostoslavsky, Gustavo, Kotton, Darrell N., Murphy, George J.
Format: Online
Language:English
Published: Elsevier 2013
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3841264/