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Direct correction of haemoglobin E β-thalassaemia using base editors.

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作者单位: MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.;Department of Clinical Haematology, Royal London Hospital, Barts Health NHS Trust, London, UK. [1] MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK. [2] MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.;State Key Laboratory of Reproductive Medicine, Nanjing Medical University, Nanjing, China. [3] MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.;MRC WIMM Centre for Computational Biology, MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK.;Oxford National Institute of Health Research Biomedical Research Centre, University of Oxford, Oxford, UK. [4] Department of Paediatrics, University of Kelaniya, Kelaniya, Sri Lanka. [5] Transplantation Research and Immunology Group, Nuffield Department of Surgical Sciences, University of Oxford, Oxford, UK. [6] MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.;Department of Paediatrics, University of Oxford, Oxford, UK. [7] Department of Clinical Haematology, Royal London Hospital, Barts Health NHS Trust, London, UK. [8] MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK.;MRC WIMM Centre for Computational Biology, MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK. [9] Laboratory of Gene Regulation, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK. [10] MRC Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, UK. james.davies@imm.ox.ac.uk.;Department of Clinical Haematology, Royal London Hospital, Barts Health NHS Trust, London, UK. james.davies@imm.ox.ac.uk.;National Institute of Health Research Blood and Transplant Research Unit in Precision Cellular Therapeutics, Oxford, UK. james.davies@imm.ox.ac.uk. [11]
DOI 10.1038/s41467-023-37604-8
PMID 37076455
发布时间 2025-02-05
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