Nature Communications (Nov 2022)
Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response
- Xiangjun He,
- Zhenjie Zhang,
- Junyi Xue,
- Yaofeng Wang,
- Siqi Zhang,
- Junkang Wei,
- Chenzi Zhang,
- Jue Wang,
- Brian Anugerah Urip,
- Chun Christopher Ngan,
- Junjiang Sun,
- Yuefeng Li,
- Zhiqian Lu,
- Hui Zhao,
- Duanqing Pei,
- Chi-Kong Li,
- Bo Feng
Affiliations
- Xiangjun He
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Zhenjie Zhang
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Junyi Xue
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Yaofeng Wang
- Centre for Regenerative Medicine and Health, Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences
- Siqi Zhang
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Junkang Wei
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Chenzi Zhang
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Jue Wang
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Brian Anugerah Urip
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Chun Christopher Ngan
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Junjiang Sun
- Division of Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina
- Yuefeng Li
- Guangdong Landau Biotechnology Co.Ltd
- Zhiqian Lu
- Department of Cardiothoracic Surgery, Shanghai Jiao Tong University Affiliated Sixth People’s Hospital
- Hui Zhao
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- Duanqing Pei
- Centre for Regenerative Medicine and Health, Hong Kong Institute of Science & Innovation, Chinese Academy of Sciences
- Chi-Kong Li
- Department of Pediatrics, Hong Kong Children’s Hospital, The Chinese University of Hong Kong
- Bo Feng
- School of Biomedical Sciences, MOE Key Lab, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong
- DOI
- https://doi.org/10.1038/s41467-022-34898-y
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 17
Abstract
Here, the authors develop an AAV-CRISPR mediated somatic knock-in. They apply this system to restore hemostasis in neonatal hemophilia B mice and show liver-specificity of the knock-in and low serum antibody production.
