Specificity of CRISPR-Cas9 Editing in Exagamglogene Autotemcel

被引：3
|
作者：
Yen, Angela [1 ]
Zappala, Zachary [1 ]
Fine, Rebecca S. [1 ]
Majarian, Timothy D. [1 ]
Sripakdeevong, Parin [2 ]
Altshuler, David [1 ]
机构：
[1] Vertex Pharmaceut, Boston, MA 02210 USA
[2] CRISPR Therapeut, Boston, MA USA
来源：
NEW ENGLAND JOURNAL OF MEDICINE | 2024年
关键词：
D O I：
10.1056/NEJMc2313119
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Potential for Off-Target Editing in Exa-cel The autologous cellular therapy exagamglogene autotemcel is generated by editing an erythroid-specific enhancer of BCL11A. Could another site be edited unintentionally? This study gauged the likelihood of off-target editing.
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页数：3
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