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CRISPR/Cas9‐mediated Somatic Correction of a Novel Coagulator Factor IX Gene Mutation Ameliorates Hemophilia in Mouse

Yuting Guan,Yanlin Ma,Qi Li,Zhenliang Sun,Lie Ma,Lijuan Wu,Liren Wang,Li Zeng,Yanjiao Shao,Yuting Chen,Ning Ma,Wenqing Lu,Kewen Hu,Honghui Han,Yanhong Yu,Yuanhua Huang,Mingyao Liu,Dali Li

EMBO Molecular Medicine（2016）

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关键词
gene therapy,genome editing,hemophilia B,hemostasis,monogenetic disease
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