CRISPR–Cas9-mediated gene editing of the BCL11A enhancer for pediatric β0/β0 transfusion-dependent β-thalassemia

作者：Bin Fu, Jiaoyang Liao, Shuanghong Chen, Wei Li, Qiudao Wang, Jian Hu, Fei Yang, Shenlin Hsiao, Yanhong Jiang, Liren Wang, Fangping Chen, Yuanjin Zhang, Xin Wang, Dali Li, Mingyao Liu, Yuxuan Wu · 发表于：Nature Medicine · 年份：2022 · DOI：10.1038/s41591-022-01906-z · 被引用次数：178 · 研究领域：Hemoglobinopathies and Related Disorders、CRISPR and Genetic Engineering、Prenatal Screening and Diagnostics