Research progress in gene therapy of hemophilia B

Hemophilia B is a common hemorrhagic disease in clinic and a X chromosome-linked recessive genetic disorder. It is common in male patients, and rare in female patients. The number of patients with hemophilia B in China is numerous, the main treatment is infusing coagulation factor FⅨ to achieve hemostasis. But coagulation factor FⅨ inhibitors caused by repeating infusion of coagulation factor FⅨ and costly price of coagulation factor FⅨ products, the majority of patients have not been effectively treated, which influence the life quality of the patients with hemophilia B. Hemophilia B is a monogenetic inherited disease and is an ideal model for genetic treatment. Therefore, gene therapy has become the most promising approach to cure hemophilia B. In recent years, with the successful construction of a variety of viral vectors and the discovery of site-specific nuclease, progress of gene therapy for hemophilia B has been further promoted. In this paper, the progress of gene therapy for hemophilia B is reviewed. Key words: Hemophilia B; Genetic therapy; Factor Ⅸ; CRISPR-Cas systems; Genetic vectors