Gene Editing of the Endogenous Cryptic 3' Splice Site Corrects the RNA Splicing Defect in the β(654)-Thalassemia Mouse Model
内源性隐蔽3'剪接位点的基因编辑可纠正β(654)-地中海贫血小鼠模型中的RNA剪接缺陷
期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2023.202
Lu, Dan; Gong, Xiuli; Guo, Xinbing; Cai, Qin; Chen, Yanwen; Zhu, Yiwen; Sang, Xiao; Yang, Hua; Xu, Miao; Zeng, Yitao; Li, Dali; Zeng, Fanyi