Chapter title |
Multisystemic Disease Modeling of Liver-Derived Protein Folding Disorders Using Induced Pluripotent Stem Cells (iPSCs).
|
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Chapter number | 194 |
Book title |
Patient-Specific Induced Pluripotent Stem Cell Models
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Published in |
Methods in molecular biology, February 2015
|
DOI | 10.1007/7651_2014_194 |
Pubmed ID | |
Book ISBNs |
978-1-4939-3033-3, 978-1-4939-3034-0
|
Authors |
Amy Leung, George J. Murphy |
Editors |
Andras Nagy, Kursad Turksen |
Abstract |
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, highlighting the need for a model capable of recapitulating the multisystem complexity of this clinically variable disease. Here, we describe detailed methodologies for the directed differentiation of protein folding disease-specific iPSCs into hepatocytes that produce mutant protein, and neural-lineage cells often targeted in disease. Methodologies are also described for the construction of multisystem models and drug screening using iPSCs. |
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