A new "FIX" for hemophilia B gene therapy

被引：3
|
作者：
Almeida-Porada, Graca [1 ]
机构：
[1] Wake Forest Sch Med, Winston Salem, NC 27101 USA
来源：
BLOOD | 2021年 / 137卷 / 21期
关键词：
FACTOR-IX;
D O I：
10.1182/blood.2021011753
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
In this issue of Blood, Nair et al(1) demonstrate that AAV-directed gene therapy using a new bioengineered FIX transgene provides higher FIX activity and superior hemostatic efficacy than other FIX variants and may allow for lower and potentially safer vector doses in future human clinical trials.
引用
收藏
页码：2860 / 2861
页数：3
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