[Gene therapy marks the beginning of a potential "clinical cure" for hemophilia B patients].

Abstract

Hemophilia B is a monogenic inherited disorder characterized by spontaneous or trauma-induced bleeding. In China, the primary treatment strategy for hemophilia B is factor Ⅸ (FⅨ) replacement therapy, requiring patients to undergo frequent venipunctures throughout their lifetime. In recent years, significant advancements have been made in gene therapy for hemophilia B. As gene therapy for hemophilia B is nearing clinical implementation in China, the Thrombosis and Hemostasis Group of the Chinese Society of Hematology of the Chinese Medical Association and the Hemophilia Treatment Center Collaborative Network of China have jointly developed the "Chinese guidance for clinical application of Adeno-associated virus vector-based gene therapy for hemophilia B (2025)", offering comprehensive recommendations for standardizing the entire gene therapy process. This article will introduce the background, key points, and clinical value of the guidelines to enhance awareness and emphasis on gene therapy for hemophilia B in China.