Research advance of gene therapy in hemophilia B
10.3760/cma.j.cn121090-20250122-00041
- VernacularTitle:基因治疗在血友病B领域的研究进展
- Author:
Tienan ZHU
1
Author Information
1. 中国医学科学院 北京协和医学院 北京协和医院血液内科，北京 100730
- Publication Type:Journal Article
- From:
Chinese Journal of Hematology
2025;46(6):588-592
- CountryChina
- Language:Chinese
-
Abstract:
Hemophilia B (HB) is an X-chromosome-linked recessive disorder caused by mutations in the gene encoding coagulation factor Ⅸ (FⅨ), resulting in coagulation factor Ⅸ deficiency. However, factor replacement treatment, as the standard of care in patients with HB at present, still faces many challenges such as the need for lifelong medication, frequent venipunctures, fluctuation of F Ⅸ activity levels after infusion, and potential risk for the development of inhibitors. Gene therapy (GT) is an approach to achieve therapeutic goals by modifying or manipulating the expression of target genes and altering the biological properties of living cells. As favorable efficacy and safety are demonstrated in various clinical trials, some GT products for HB have been successfully marketed, and the clinical treatment landscape of HB is facing important changes. This article summarizes the major advances in this field and discusses some noteworthy issues to provide references for relevant research and clinical practice.
