TY - JOUR
T1 - Contributions of animal models to the mechanisms and therapies of transthyretin amyloidosis
AU - Ibrahim, Ridwan Babatunde
AU - Liu, Yo Tsen
AU - Yeh, Ssu Yu
AU - Tsai, Jin Wu
N1 - Publisher Copyright:
© 2019 Ibrahim, Liu, Yeh and Tsai.
PY - 2019
Y1 - 2019
N2 - 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.
AB - 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.
KW - Animal model
KW - C. elegans
KW - D. melanogaster
KW - Familial amyloid polyneuropathy
KW - IPSC
KW - Neurodegenerative disorder
KW - Transgenic mouse
KW - Transthyretin
UR - http://www.scopus.com/inward/record.url?scp=85068235166&partnerID=8YFLogxK
U2 - 10.3389/fphys.2019.00338
DO - 10.3389/fphys.2019.00338
M3 - Review article
AN - SCOPUS:85068235166
SN - 1664-042X
VL - 10
JO - Frontiers in Physiology
JF - Frontiers in Physiology
IS - APR
M1 - 338
ER -