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,...
Main Authors: | , , , , , , , , , , , , , , |
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Format: | Online |
Language: | English |
Published: |
Elsevier
2013
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Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3841264/ |