Genes and Diseases (Jan 2024)
Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs
- Fei Yang,
- Shuqian Xu,
- Chao Huang,
- Zhiqiang Shao,
- Yuefen Hu,
- Yang Yang,
- Yongrong Lai,
- Qing Ke,
- Dan Liang,
- Yuxuan Wu
Affiliations
- Fei Yang
- Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China; BRL Medicine Inc., Shanghai 201108, China
- Shuqian Xu
- Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong 250012, China
- Chao Huang
- BRL Medicine Inc., Shanghai 201108, China
- Zhiqiang Shao
- BRL Medicine Inc., Shanghai 201108, China
- Yuefen Hu
- Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, Shandong 250012, China
- Yang Yang
- Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi 530000, China
- Yongrong Lai
- Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi 530000, China
- Qing Ke
- Department of Neurology, The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, Zhejiang 310003, China
- Dan Liang
- 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; Corresponding author. Department of Obstetrics and Gynecology, The First Affiliated Hospital of Anhui Medical University, Hefei, Anhui 230022, China.
- Yuxuan Wu
- Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China; BRL Medicine Inc., Shanghai 201108, China; Corresponding author. Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai 200241, China.
- Journal volume & issue
-
Vol. 11,
no. 1
pp. 15 – 18
