Correction of β-thalassemia mutant by base editor in human embryos

• Published in: Protein & cell Vol. 8; no. 11; pp. 811 - 822
• Main Authors: Liang, Puping, Ding, Chenhui, Sun, Hongwei, Xie, Xiaowei, Xu, Yanwen, Zhang, Xiya, Sun, Ying, Xiong, Yuanyan, Ma, Wenbin, Liu, Yongxiang, Wang, Yali, Fang, Jianpei, Liu, Dan, Songyang, Zhou, Zhou, Canquan, Huang, Junjiu
• Format: Journal Article
• Language: English
• Published: Beijing Higher Education Press 01.11.2017; Springer Nature B.V; Oxford University Press
• Subjects: APOBEC-1 Deaminase - genetics; APOBEC-1 Deaminase - metabolism; base editor; Base Sequence; beta-Globins - genetics; beta-Globins - metabolism; beta-Thalassemia - genetics; beta-Thalassemia - metabolism; beta-Thalassemia - pathology; beta-Thalassemia - therapy; Biochemistry; Biomedical and Life Sciences; Blastomeres; Blastomeres - cytology; Blastomeres - metabolism; Cell Biology; CRISPR-Cas Systems; Deamination; Developmental Biology; Embryo, Mammalian - metabolism; Embryo, Mammalian - pathology; Feasibility studies; Female; Fibroblasts; Fibroblasts - metabolism; Fibroblasts - pathology; Gene Editing - methods; Gene Expression; HBB gene; HBB −28 (A>G); HEK293 Cells; Heterozygote; Homozygote; human embryo; Human Genetics; Humans; Life Sciences; Mutation; Nuclear transfer; Oocytes; Point Mutation; Primary Cell Culture; Promoter Regions, Genetic; Protein Science; Sequence Analysis, DNA; Short; Short Article; Skin; Somatic cells; Stem Cells; Thalassemia; β-thalassemia; 28 (A>G); base editor; human embryo; β-thalassemia; HBB −28 (A>G)

β-Thalassemia is a global health issue, caused by mutations in the HBB gene. Among these mutations, HBB -28 （A〉G） mutations is one of the three most common mutations in China and Southeast Asia patients with β-thalassemia. Correcting this mutation in human embryos may prevent the disease being passe...