Transthyretin Amyloidosis (ATTR) Models
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Transthyretin amyloidosis (ATTR) is a rare disease characterized by the abnormal accumulation of amyloid deposits made up of misfolded transthyretin (TTR) proteins in various organs, including the retina, vitreous, kidneys, and brain. Two prominent subtypes of ATTR are transthyretin amyloid polyradiculoneuropathy (ATTR-PN) and transthyretin amyloid cardiomyopathy (ATTR-CM).
GemPharmatech has developed three mouse models by gene-editing technology. These models can be utilized for studying ATTR and evaluating the in vivo efficacy of human TTR-targeted gene and other nucleic acid-based therapies.
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Strain No.
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Strain Name | Strain Type | Description |
|---|---|---|---|
| T059956 | B6-hTTR V30M Tg mTtr-KO | Transgenic | Mice were developed using gene editing technology to transfer the human TTR gene with V30M mutation by transgenic method on the basis of knockout of mouse Ttr gene. Human TTR protein expression can be detected in mouse serum, while mouse TTR protein is not expressed. |
| T059897 | B6-hTTR-V30M | Transgenic | The human TTR gene with V30M mutation were transferred into mouse genome to generate B6-hTTR V30M Tg mouse model. Human TTR protein expression can be detected in the serum of mice at 8 weeks of age. |
| T055186 | B6-hTTR | KI | The coding region and regulatory sequences of the murine TTR gene were replaced with the corresponding human gene fragments to generate B6-hTTR mouse model. Human TTR protein expression can be detected in the serum of mice at 5 months of age. |