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

doi:10.1016/j.gendis.2023.01.010

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

Short communication | 更新时间：2024-02-04

- Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs
- Genes & Diseases Vol. 1, Issue 1, Pages: 15-18(2024)
- 作者机构：
  
  1. Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University,Shanghai,China,200241
  2. BRL Medicine Inc.,Shanghai,China,201108
  3. Department of Hematology, Qilu Hospital, Cheeloo College of Medicine, Shandong University,Shandong,Jinan,China,250012
  4. Department of Hematology, The First Affiliated Hospital of Guangxi Medical University,Guangxi,Nanning,China,530000
  5. Department of Neurology, The First Affiliated Hospital, Zhejiang University School of Medicine,Zhejiang,Hangzhou,China,310003
  6. Department of Obstetrics and Gynecology, The First Affiliated Hospital of Anhui Medical University,Anhui,Hefei,China,230022
  7. NHC Key Laboratory of Study on Abnormal Gametes and Reproductive Tract (Anhui Medical University),Anhui,Hefei,China,230032
- 作者简介：
- 基金信息：
- DOI：10.1016/j.gendis.2023.01.010
  CLC：
- Published：2024，
  
  Published Online：1 January 2024，
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Fei Yang, Shuqian Xu, Chao Huang, et al. Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs. [J]. Genes & Diseases 1(1):15-18(2024)
DOI：

Fei Yang, Shuqian Xu, Chao Huang, et al. Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs. [J]. Genes & Diseases 1(1):15-18(2024) DOI： 10.1016/j.gendis.2023.01.010.

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1 T.C. Cheng, S.H. Orkin, S.E. Antonarakis, et al.Beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects Proc Natl Acad Sci U S A, 81 (9) (1984), pp. 2821-2825

2 S. Xu, K. Luk, Q. Yao, et al.Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia Blood, 133 (21) (2019), pp. 2255-2262

3 B.E. McIntosh, M.E. Brown, B.M. Duffin, et al.Nonirradiated NOD, B6.SCID Il2rγ−/− KitW41/W41 (NBSGW) mice support multilineage engraftment of human hematopoietic cells Stem Cell Rep, 4 (2) (2015), pp. 171-180

4 S. Bae, J. Park, J.S. KimCas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases Bioinformatics, 30 (10) (2014), pp. 1473-1475

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