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Article

A library of ATTR amyloidosis patient-specific induced pluripotent stem cells for disease modelling and in vitro testing of novel therapeutics.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis - 1 Sept 2018

Giadone Richard M, Rosarda Jessica D, Akepati Prithvi Reddy, Thomas Arianne C, Boldbaatar Batbold, James Marianne F, Wilson Andrew A, Sanchorawala Vaishali, Connors Lawreen H, Berk John L, Wiseman R Luke, Murphy George J

Abstract excerpt

Hereditary transthyretin amyloidosis (ATTR amyloidosis) is an autosomal dominant protein-folding disorder caused by over 100 distinct mutations in the transthyretin (TTR) gene. In ATTR amyloidosis, protein secreted from the liver aggregates and forms amyloid fibrils in downstream target organs, chiefly the heart and peripheral nervous system. Few animal models of ATTR amyloidosis exist and none recapitulate the...

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Identifiers

PMID: 30032658
DOI: 10.1080/13506129.2018.1489228
PMCID: PMC6319917

Genes

TTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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