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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,...
Autores principales: | 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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2013
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3841264/ https://www.ncbi.nlm.nih.gov/pubmed/24286032 http://dx.doi.org/10.1016/j.stemcr.2013.10.003 |
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