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

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

*此作品的通信作者

研究成果: Review article同行評審

10 引文 斯高帕斯(Scopus)

摘要

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.

原文English
文章編號338
期刊Frontiers in Physiology
10
發行號APR
DOIs
出版狀態Published - 2019

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