Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs
利用切口酶将功能增强的FVIII基因整合到患者来源的iPSCs的AAVS1基因座中,从而实现A型血友病的长期矫正
期刊:Experimental and Molecular Medicine
影响因子:12.9
doi:10.1038/s12276-024-01375-z
Kim, Do-Hun; Choi, Sang-Hwi; Sung, Jin Jea; Kim, Sieun; Yi, Hanui; Park, Sanghyun; Park, Chan Wook; Oh, Young Woo; Lee, Jungil; Kim, Dae-Sung; Kim, Jong-Hoon; Park, Chul-Yong; Kim, Dong-Wook