Mouse models of transthyretin amyloidosis

Abstract

Transthyretin (TTR) amyloidosis is caused by mutations in the TTR gene. Several observations, however, suggest the presence of factors, other than a mutation in the TTR gene, which affect TTR amyloid deposition. Although liver transplantation is the only curative treatment for TTR amyloidosis, its donor pool faces shortage, and TTR amyloid deposition continues in many patients after transplantation. Thus, some effective therapeutic strategies other than liver transplantation need to be developed. Mouse models of TTR amyloidosis would facilitate defining factors that accelerate amyloid deposition and would aid in developing effective treatments. Here, we summarize studies of transgenic mouse models of TTR amyloidosis in which questions were addressed about the role of various risk factors in the molecular pathogenesis of this intractable disease. © 2009 Springer-Verlag Berlin Heidelberg.