Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs

作者:Yang, Fei; Xu, Shuqian; Huang, Chao; Shao, Zhiqiang; Hu, Yuefen; Yang, Yang; Lai, Yongrong; Ke, Qing; Liang, Dan*; Wu, Yuxuan*
来源:Genes & Diseases, 2024, 11(1): 15-18.
DOI:10.1016/j.gendis.2023.01.010

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