Article
CRISPR/Cas9-mediated β-globin gene knockout in rabbits recapitulates human β-thalassemia.
The Journal of biological chemistry - 1 Jan 2000
Yang Yi, Kang Xiangjin, Hu Shiqi, Chen Bangzhu, Xie Yingjun, Song Bing, Zhang Quanjun, Wu Han, Ou Zhanhui, Xian Yexing, Fan Yong, Li Xiaoping, Lai Liangxue, Sun Xiaofang
Abstract excerpt
β-thalassemia, an autosomal recessive blood disorder that reduces the production of hemoglobin, is majorly caused by the point mutation of the HBB gene resulting in reduced or absent β-globin chains of the hemoglobin tetramer. Animal models recapitulating both the phenotype and genotype of human disease are valuable in the exploration of pathophysiology and for in vivo evaluation of novel therapeutic treatments....
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