# Gene therapy marks the beginning of a potential “clinical cure” for hemophilia B patients

## TL;DR

This paper discusses gene therapy as a potential clinical cure for hemophilia B, highlighting new guidelines for its clinical application in China.

## Contribution

The paper introduces the first clinical application guidelines for AAV-based gene therapy for hemophilia B in China.

## Key findings

- Gene therapy is emerging as a potential clinical cure for hemophilia B.
- New guidelines provide detailed recommendations for the clinical application of gene therapy in China.

## Abstract

血友病B是一种单基因遗传病，主要表现为全身各部位的自发性或外伤后出血。当前国内对于血友病B的治疗策略主要是因子替代治疗，患者终身面临频繁静脉穿刺。近年来，血友病B基因疗法发展迅速。在血友病B基因疗法即将进入中国临床之际，中华医学会血液学分会血栓与止血学组和中国血友病协作组联合制定了《中国血友病B腺相关病毒载体基因治疗临床应用指导意见（2025）》，为血友病B基因疗法全流程的规范化给出了详细的指导意见。本文将针对《指导意见》形成的背景、核心要点和临床价值做介绍，借以提高国内对基因治疗的认知和重视度。