[HTML][HTML] Amyloid deposition in a mouse model humanized at the transthyretin and retinol-binding protein 4 loci
X Li, Y Lyu, J Shen, Y Mu, L Qiang, L Liu, K Araki… - Laboratory …, 2018 - Elsevier
Familial amyloidotic polyneuropathy is an autosomal dominant disorder caused by a point
mutation in the transthyretin (TTR) gene. The process of TTR amyloidogenesis begins with
rate-limiting dissociation of the TTR tetramer. Thus, the TTR stabilizers, such as Tafamidis
and Diflunisal, are now in clinical trials. Mouse models will be useful to testing the efficacy of
these drugs. Although several mouse models have been generated, they all express mouse
Rbp4. Thus, human TTR associates with mouse RBP4, resulting in different kinetic and …
mutation in the transthyretin (TTR) gene. The process of TTR amyloidogenesis begins with
rate-limiting dissociation of the TTR tetramer. Thus, the TTR stabilizers, such as Tafamidis
and Diflunisal, are now in clinical trials. Mouse models will be useful to testing the efficacy of
these drugs. Although several mouse models have been generated, they all express mouse
Rbp4. Thus, human TTR associates with mouse RBP4, resulting in different kinetic and …
