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2016 1

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AAV8 1

gene therapy 1

hFIX 1

hemophilia B 1

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Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX

Wei Lu,Qingzhang Zhou,Hao Yang,Hao Wang,Yexing Gu,Qi Shen,Jinglun Xue,Xiaoyan Dong,Jinzhong Chen

Frontiers of Medicine 2016, Volume 10, Issue 2,   Pages 212-218 doi: 10.1007/s11684-016-0438-y

Abstract: into plasmid scAAV8-LP1 to develop an optimized AAV8 vector that expresses human clotting factor FIX (hFIXThe efficiency of scAAV8-LP1-hFIX administered through normal systemic injection or hydrodynamic injectionA high expression was achieved using hydrodynamic injection, and the peak hFIX expression levels in the

Keywords： hemophilia B     AAV8     hFIX     gene therapy    

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Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX

Wei Lu,Qingzhang Zhou,Hao Yang,Hao Wang,Yexing Gu,Qi Shen,Jinglun Xue,Xiaoyan Dong,Jinzhong Chen

Journal Article