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

Genes Dis. 2023 Mar 24;11(1):15-18. doi: 10.1016/j.gendis.2023.01.010. eCollection 2024 Jan.

Fei Yang^{1

2}, Shuqian Xu³, Chao Huang², Zhiqiang Shao², Yuefen Hu³, Yang Yang⁴, Yongrong Lai⁴, Qing Ke⁵, Dan Liang^{6

7}, Yuxuan Wu^{1

2}

¹ Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China.
² BRL Medicine Inc., Shanghai 201108, China.
³ Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong 250012, China.
⁴ Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi 530000, China.
⁵ Department of Neurology, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310003, China.
⁶ Department of Obstetrics and Gynecology, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui 230022, China.
⁷ NHC Key Laboratory of Study on Abnormal Gametes and Reproductive Tract (Anhui Medical University), Hefei, Anhui 230032, China.

No abstract available