Article
Treatment of β654 -thalassaemia by TALENs in a mouse model.
Cell proliferation - 1 Dec 2018
Fang Yudan, Cheng Yan, Lu Dan, Gong Xiuli, Yang Guanheng, Gong Zhijuan, Zhu Yiwen, Sang Xiao, Fan Shuyue, Zhang Jingzhi, Zeng Fanyi
Abstract excerpt
OBJECTIVES: This study explored whether TALENs-mediated non-homologous end joining (NHEJ) targeting the mutation site can correct the aberrant β-globin RNA splicing, and ameliorate the β-thalassaemia phenotype in β654 mice. MATERIAL AND METHODS: TALENs vectors targeted to the human β-globin gene (HBB) IVS2-654C >T mutation in a mouse model were constructed and selected to generate double heterozygous TALENs+...
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