The Guard1 clinical trial - A first in-human, phase 1/2 study evaluating AVR-RD-02, a hematopoietic stem cell (HSC) gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion

被引：1
|
作者：
Adera, Mathews [1 ]
Veselinovic, Milena [1 ]
Treohan, Anita [2 ]
Golipour, Azadeh [3 ]
Ridha, Essra [1 ]
机构：
[1] AVROBIO Inc, Clin Dev, Cambridge, MA USA
[2] AVROBIO Inc, Clin Dev Operat, Cambridge, MA USA
[3] AVROBIO Inc, CMC, Cambridge, MA USA
来源：
MOLECULAR GENETICS AND METABOLISM | 2023年 / 138卷 / 02期
关键词：
D O I：
10.1016/j.ymgme.2022.106991
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
8
引用
收藏
页码：3 / 4
页数：2
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