From GWAS To the Clinic: Genome-Editing the Human BCL11A Erythroid Enhancer for Fetal Globin Elevation in the Hemoglobinopathies

被引：0
|
作者：
Reik, Andreas [1 ]
Chang, Kai-Hsin [2 ]
Vierstra, Jeff [2 ]
Psatha, Nikoletta [2 ]
Stehling-Sun, Sandra [2 ]
Zhou, Yuanyue [1 ]
O'Neil, Colleen M. [1 ]
Mich, Andrea [1 ]
Miller, Jeffrey C. [1 ]
Lee, Gary [1 ]
Tan, Siyuan [3 ]
Jiang, Haiyan [3 ]
Stamatoyannopoulos, George [2 ]
Papayannopoulou, Thalia [2 ]
Rebar, Edward J. [1 ]
Gregory, Philip D. [1 ]
Stamatoyannopoulos, John A. [2 ]
Urnov, Fyodor D. [1 ]
机构：
[1] Sangamo BioSci Inc, Richmond, CA USA
[2] Univ Washington, Seattle, WA 98195 USA
[3] Biogen Idec Inc, Cambridge, MA USA
来源：
MOLECULAR THERAPY | 2015年 / 23卷
关键词：
D O I：
10.1016/S1525-0016(16)33658-9
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
53
引用
收藏
页码：S23 / S24
页数：2
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