Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs.
作者信息
Yang Fei, Xu Shuqian, Huang Chao, Shao Zhiqiang, Hu Yuefen, Lai Yongrong, Ke Qing, Liang Dan, Wu Yuxuan
机构信息
Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China.
BRL Medicine Inc., Shanghai 201108, China.
出版信息
Genes Dis. 2023 Mar 24;11(1):15-18. doi: 10.1016/j.gendis.2023.01.010. eCollection 2024 Jan.
Abstract
摘要
Zotero 插件