In vivo generated hematopoietic stem cells from genome edited induced pluripotent stem cells are functional in platelet-targeted gene therapy of murine hemophilia A

• Dawei Wang,
• Guowei Zhang,
• Junjie Gu,
• Xiaohu Shao,
• Yuting Dai,
• Jianfeng Li,
• Xiaohong Pan,
• Shuxian Yao,
• Aining Xu,
• Ying Jin,
• Jinyan Huang,
• Qizhen Shi,
• Jiang Zhu,
• Xiaodong Xi,
• Zhu Chen,
• Saijuan Chen

Affiliations

Dawei Wang

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China;National Research Center for Translational Medicine, Shanghai Jiao Tong University School of Medicine, Shanghai, China

Guowei Zhang

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China;Key Laboratory of Aging and Cancer Biology of Zhejiang Province, Hangzhou Normal University School of Medicine, Hangzhou, China

Junjie Gu

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

Xiaohu Shao

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Yuting Dai

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Jianfeng Li

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Xiaohong Pan

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Shuxian Yao

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Aining Xu

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Ying Jin

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

Jinyan Huang

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Qizhen Shi

Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA

Jiang Zhu

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Xiaodong Xi

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China

Zhu Chen

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China;National Research Center for Translational Medicine, Shanghai Jiao Tong University School of Medicine, Shanghai, China

Saijuan Chen

State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China;National Research Center for Translational Medicine, Shanghai Jiao Tong University School of Medicine, Shanghai, China