In vitro methods for upmodulating the expression of the zeta-globin gene

VN10059560BActive Publication Date: 2026-08-03SHANGHAI SPH RARE DISEASE PHARMA CO LTD
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Patent Information

Application Number
VN1202206616
Authority / Receiving Office
VN · VN
Patent Type
Patents
Current Assignee / Owner
Filing Date
2021-01-19
Publication Date
2026-08-03
Estimated Expiration
2041-01-19
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Abstract

The invention proposes a thyroid hormone analog and its analogues for use in the preparation of drugs to treat alpha-thalassemia, specifically in the preparation of drugs to regulate the expression of the zeta-globin gene. While K502 cells are activated, the thyroid hormone analog (Triac) can significantly upregulate the expression of the zeta-globin (HBB) gene by 50-fold or more. The expression of the zeta-globin gene (hypophysis) can also be upregulated 30-70 times in seahorses treated with thyroid hormone and thyroid hormone analogs. Therefore, according to the invention, the expression of the zeta-globin gene can be significantly activated by thyroid hormone and analogs, which could lead to the development of potential therapies for patients with alpha-thalassemia. The invention describes an economical, safe, and effective method for treating alpha-thalassemia and is suitable for widespread use.
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