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Rapid Communication | Open Access

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

Fei Yanga,e,1, Shuqian Xud,1, Chao Huange, Zhiqiang Shaoe, Yuefen Hud, Yang Yangf, Yongrong Laif, Qing Keg, Dan Liangb,c( ), Yuxuan Wua,e( )
Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China
Department of Obstetrics and Gynecology, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui 230022, China
NHC Key Laboratory of Study on Abnormal Gametes and Reproductive Tract (Anhui Medical University), Hefei, Anhui 230032, China
Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong 250012, China
BRL Medicine Inc., Shanghai 201108, China
Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi 530000, China
Department of Neurology, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310003, China

Peer review under responsibility of Chongqing Medical University.

1 These authors contributed equally to this work.

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Genes & Diseases
Volume 11 Issue 1,
January 2024
Pages 15-18
DOI: 10.1016/j.gendis.2023.01.010

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Cite this article:
Yang F, Xu S, Huang C, et al. Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs. Genes & Diseases, 2024, 11(1): 15-18. https://doi.org/10.1016/j.gendis.2023.01.010

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Received: 19 October 2022
Published: 24 March 2023
© 2023 The Authors.

This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).