# Research advance of gene therapy in hemophilia B

## TL;DR

This paper reviews recent advances in gene therapy for hemophilia B, a genetic disorder caused by a deficiency in clotting factor IX.

## Contribution

The paper summarizes key developments in gene therapy for hemophilia B and discusses important challenges and future directions.

## Key findings

- Gene therapy has shown promising efficacy and safety in clinical trials for hemophilia B.
- Some gene therapy products for hemophilia B are already approved or close to market release.
- Gene therapy could potentially replace traditional lifelong treatment methods for the disease.

## Abstract

血友病B（Hemophilia B, HB）是一种X染色体连锁隐性遗传病，因编码凝血因子Ⅸ（FⅨ）的基因突变引起凝血因子Ⅸ缺乏所致。替代治疗为目前的标准治疗措施，但面临着需终身用药、频繁静脉穿刺、输注后FⅨ活性水平存在波峰波谷影响疗效以及可能产生抑制物等诸多挑战。基因治疗是指通过基因替代或编辑技术，恢复改变功能细胞的生物学特性，以达到治疗目的的医学手段。随着多个临床试验所展现的良好疗效和安全性，针对HB的部分基因治疗产品已成功或即将上市，HB的临床治疗格局面临重要变化。本文梳理了这一领域的主要进展，并对一些重要问题加以讨论，为相关研究和实践提供参考。