Clinical applications of gene editing and therapy
Mohsin Badat
Centre for Genomics and Child Health, Blizard Institute, Queen Mary University of London, UK
Search for more papers by this authorJames Davies
MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK
National Institute of Health Research Blood and Transplant Research Unit in Precision Cellular Therapeutics, Oxford, UK
Search for more papers by this authorMohsin Badat
Centre for Genomics and Child Health, Blizard Institute, Queen Mary University of London, UK
Search for more papers by this authorJames Davies
MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK
National Institute of Health Research Blood and Transplant Research Unit in Precision Cellular Therapeutics, Oxford, UK
Search for more papers by this authorAdam J Mead PhD, FRCP, FRCPath, FMedSci
Haematopoietic Stem Cell Biology Laboratory, Medical Research Council Molecular Haematology Unit, Medical Research Council Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK
Search for more papers by this authorMichael A Laffan DM, MRCP, FRCPath
Department of Immunology and Inflammation, Faculty of Medicine, Imperial College London, London, UK
Search for more papers by this authorGraham P Collins DPhil, FRCP, FRCPath
Department of Haematology, Oxford Cancer and Haematology Centre, Oxford, UK
Search for more papers by this authorDeborah Hay DPhil, MRCP, FRCPath
Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford, UK
Search for more papers by this authorA Victor Hoffbrand MA, DM, FRCP, FRCPath, FRCP (Edin), DSc, FMedSci
Emeritus Professor of Haematology Honorary Consultant Haematologist
University College London, London, UK
Royal Free Hospital, London, UK
Search for more papers by this authorSummary
Haematology is at the forefront of the clinical development of advanced genetic therapies. This field is rapidly progressing as evidenced by the recent regulatory approval of adeno-associated virus (AAV)-based therapies for Haemophilia A and B and CRISPR-Cas9-based genome editing for thalassaemia and sickle cell disease. Providing the safety and health economics of these approaches are established, it is likely that these advanced therapeutics will become the standard of care in the future for many patients with inherited haematological diseases.
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