Contributions of animal models to the mechanisms and therapies of transthyretin amyloidosis

Ridwan Babatunde Ibrahim, Yo Tsen Liu, Ssu Yu Yeh, Jin Wu Tsai*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

10 Scopus citations

Abstract

Transthyretin amyloidosis (ATTR amyloidosis) is a fatal systemic disease caused by amyloid deposits of misfolded transthyretin, leading to familial amyloid polyneuropathy and/or cardiomyopathy, or a rare oculoleptomeningeal amyloidosis. A good model system that mimic the disease phenotype is crucial for the development of drugs and treatments for this devastating degenerative disorder. The present models using fruit flies, worms, rodents, non-human primates and induced pluripotent stem cells have helped researchers understand important disease-related mechanisms and test potential therapeutic options. However, the challenge of creating an ideal model still looms, for these models did not recapitulates all symptoms, particularly neurological presentation, of ATTR amyloidosis. Recently, knock-in techniques was used to generate two humanized ATTR mouse models, leading to amyloid deposition in the nerves and neuropathic manifestation in these models. This review gives a recent update on the milestone, progress, and challenges in developing different models for ATTR amyloidosis research.

Original languageEnglish
Article number338
JournalFrontiers in Physiology
Volume10
Issue numberAPR
DOIs
StatePublished - 2019

Keywords

  • Animal model
  • C. elegans
  • D. melanogaster
  • Familial amyloid polyneuropathy
  • IPSC
  • Neurodegenerative disorder
  • Transgenic mouse
  • Transthyretin

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