Transthyretin amyloidosis (ATTR) is a rare disease caused by the abnormal accumulation of amyloid deposits composed of misfolded transthyretin (TTR) proteins in multiple organs of the body, including the retina, the vitreous, the kidney, and the brain. Transthyretin amyloid polyradiculoneuropathy (ATTR-PN) and transthyretin amyloid cardiomyopathy (ATTR-CM) are prominent subtypes.
GemPharmatech replaces the coding region and regulatory sequences of the murine TTR gene with the corresponding human gene fragments to generate B6-hTTR and B6-hTTR V50M models, which can be used for the study of ATTR and the in vivo efficacy evaluation of human TTR-targeted gene therapies.
|Strain Name||Strain Type||Description|
|T055186||B6-hTTR||Cas9-KI||GemPharmatech has established the B6-hTTR model, in which human TTR has been inserted at the region of mouse Ttr. This model can be used to evaluate the efficacy of gene therapies and other drugs in vivo.|
|T054363||B6-H11-Alb-hTTR V50M||Cas9-KI||GemPharmatech established the B6-H11-Alb-hTTR V50M model which expresses human TTR with the point mutation V50M in the liver. This model can be used to evaluate the efficacy of gene therapies and other drugs in vivo.|