In vivo generated hematopoietic stem cells from genome edited induced pluripotent stem cells are functional in platelet-targeted gene therapy of murine hemophilia A
1 State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
2 National Research Center for Translational Medicine, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
3 State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China gzhang@hznu.edu.cn.
4 Key Laboratory of Aging and Cancer Biology of Zhejiang Province, Hangzhou Normal University School of Medicine, Hangzhou, China.
5 Key Laboratory of Stem Cell Biology, Center for The Excellence in Molecular and Cell Sciences, Institute of Health Sciences, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences and Shanghai Jiao Tong University School of Medicine, Shanghai, China.
6 Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA.
7 State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China sjchen@stn.sh.cn.