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Successful AAV8 gene therapy on hepatic ex situ machine perfusion for mitochondrial neurogastrointestinal encephalomyopathy.

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Title: Successful AAV8 gene therapy on hepatic ex situ machine perfusion for mitochondrial neurogastrointestinal encephalomyopathy.
Authors: Brevini T; Cambridge Stem Cell Institute, Cambridge, UK., Swift L; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK., Reynolds H; Cambridge Stem Cell Institute, Cambridge, UK., Ong J; Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Stopforth RJ; Cambridge Institute of Therapeutic Immunology & Infectious Disease (CITIID), Department of Medicine, University of Cambridge, Cambridge, UK., Malam Y; Cambridge Stem Cell Institute, Cambridge, UK., Spiers HVM; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK., Jovanovic E; Cambridge Stem Cell Institute, Cambridge, UK., Scaravaglio M; Cambridge Stem Cell Institute, Cambridge, UK; Department of Medicine and Surgery, University of Milano-Bicocca, Italy., Vakeeswarasarma V; Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Heslop J; Cambridge Stem Cell Institute, Cambridge, UK., Emmett E; Synnovis Biochemical Sciences, St Thomas' Hospital, London, UK., Andreatta B; Cambridge University Hospitals NHS Foundation Trust, Cambridge, Cambridgeshire, UK., Athanasiadis E; Cambridge Stem Cell Institute, Cambridge, UK; Medical Image and Signal Processing Laboratory, Department of Biomedical Engineering, University of West Attica, Athens, Greece., Cacciottolo TM; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; University of Cambridge Metabolic Research Laboratories, Institute of Metabolic Science, Cambridge, UK., Fuchs CD; Hans Popper Laboratory of Molecular Hepatology, Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria., Trauner M; Hans Popper Laboratory of Molecular Hepatology, Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria., Hosgood SA; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK., See TC; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Pantelis P; Department of Histology and Embryology, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece., Foukaneli D; Cambridge University Hospitals NHS Foundation Trust, Cambridge, Cambridgeshire, UK., Gelson WTH; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Gibbs P; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Allinson K; Department of Histopathology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Pettigrew GJ; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK., Duckworth A; Department of Histopathology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Paterson AL; Department of Histopathology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Gorgoulis VG; Department of Histology and Embryology, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece; Ninewells Hospital and Medical School, University of Dundee, Dundee, UK; Biomedical Research Foundation, Academy of Athens, Athens, Greece., Thompson RJ; Institute of Liver Studies, King's College, London, United Kingdom., Kaser A; Cambridge Institute of Therapeutic Immunology & Infectious Disease (CITIID), Department of Medicine, University of Cambridge, Cambridge, UK., Kosmoliaptsis V; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK., Chinnery PF; Department of Clinical Neurosciences and MRC Mitochondrial Biology Unit, University of Cambridge, UK., Marti R; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, Spain; Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain., van den Ameele J; Department of Clinical Neurosciences and MRC Mitochondrial Biology Unit, University of Cambridge, UK., Webb GJ; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Watson CJE; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK., Butler AJ; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK., Sampaziotis F; Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK; Department of Medicine, University of Cambridge, Cambridge, UK; Centre for Translational Stem Cell Biology, Hong Kong, China. Electronic address: fs347@cam.ac.uk.
Source: Journal of hepatology [J Hepatol] 2025 Nov; Vol. 83 (5), pp. 1218-1225. Date of Electronic Publication: 2025 Aug 06.
Publication Type: Case Reports; Journal Article
Journal Info: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 8503886 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1600-0641 (Electronic) Linking ISSN: 01688278 NLM ISO Abbreviation: J Hepatol Subsets: MEDLINE
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  Data: Successful AAV8 gene therapy on hepatic ex situ machine perfusion for mitochondrial neurogastrointestinal encephalomyopathy.
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  Data: <searchLink fieldCode="AU" term="%22Brevini+T%22">Brevini T</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Swift+L%22">Swift L</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Reynolds+H%22">Reynolds H</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Ong+J%22">Ong J</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Stopforth+RJ%22">Stopforth RJ</searchLink>; Cambridge Institute of Therapeutic Immunology & Infectious Disease (CITIID), Department of Medicine, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Malam+Y%22">Malam Y</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Spiers+HVM%22">Spiers HVM</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Jovanovic+E%22">Jovanovic E</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Scaravaglio+M%22">Scaravaglio M</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK; Department of Medicine and Surgery, University of Milano-Bicocca, Italy.<br /><searchLink fieldCode="AU" term="%22Vakeeswarasarma+V%22">Vakeeswarasarma V</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Heslop+J%22">Heslop J</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Emmett+E%22">Emmett E</searchLink>; Synnovis Biochemical Sciences, St Thomas' Hospital, London, UK.<br /><searchLink fieldCode="AU" term="%22Andreatta+B%22">Andreatta B</searchLink>; Cambridge University Hospitals NHS Foundation Trust, Cambridge, Cambridgeshire, UK.<br /><searchLink fieldCode="AU" term="%22Athanasiadis+E%22">Athanasiadis E</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK; Medical Image and Signal Processing Laboratory, Department of Biomedical Engineering, University of West Attica, Athens, Greece.<br /><searchLink fieldCode="AU" term="%22Cacciottolo+TM%22">Cacciottolo TM</searchLink>; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; University of Cambridge Metabolic Research Laboratories, Institute of Metabolic Science, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Fuchs+CD%22">Fuchs CD</searchLink>; Hans Popper Laboratory of Molecular Hepatology, Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria.<br /><searchLink fieldCode="AU" term="%22Trauner+M%22">Trauner M</searchLink>; Hans Popper Laboratory of Molecular Hepatology, Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria.<br /><searchLink fieldCode="AU" term="%22Hosgood+SA%22">Hosgood SA</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22See+TC%22">See TC</searchLink>; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Pantelis+P%22">Pantelis P</searchLink>; Department of Histology and Embryology, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece.<br /><searchLink fieldCode="AU" term="%22Foukaneli+D%22">Foukaneli D</searchLink>; Cambridge University Hospitals NHS Foundation Trust, Cambridge, Cambridgeshire, UK.<br /><searchLink fieldCode="AU" term="%22Gelson+WTH%22">Gelson WTH</searchLink>; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Gibbs+P%22">Gibbs P</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Allinson+K%22">Allinson K</searchLink>; Department of Histopathology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Pettigrew+GJ%22">Pettigrew GJ</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Duckworth+A%22">Duckworth A</searchLink>; Department of Histopathology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Paterson+AL%22">Paterson AL</searchLink>; Department of Histopathology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Gorgoulis+VG%22">Gorgoulis VG</searchLink>; Department of Histology and Embryology, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece; Ninewells Hospital and Medical School, University of Dundee, Dundee, UK; Biomedical Research Foundation, Academy of Athens, Athens, Greece.<br /><searchLink fieldCode="AU" term="%22Thompson+RJ%22">Thompson RJ</searchLink>; Institute of Liver Studies, King's College, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Kaser+A%22">Kaser A</searchLink>; Cambridge Institute of Therapeutic Immunology & Infectious Disease (CITIID), Department of Medicine, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Kosmoliaptsis+V%22">Kosmoliaptsis V</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Chinnery+PF%22">Chinnery PF</searchLink>; Department of Clinical Neurosciences and MRC Mitochondrial Biology Unit, University of Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Marti+R%22">Marti R</searchLink>; Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, Spain; Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Barcelona, Catalonia, Spain.<br /><searchLink fieldCode="AU" term="%22van+den+Ameele+J%22">van den Ameele J</searchLink>; Department of Clinical Neurosciences and MRC Mitochondrial Biology Unit, University of Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Webb+GJ%22">Webb GJ</searchLink>; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Watson+CJE%22">Watson CJE</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Butler+AJ%22">Butler AJ</searchLink>; Department of Surgery, University of Cambridge and NIHR Cambridge Biomedical Research Centre, Cambridge, UK; Roy Calne Transplant Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Sampaziotis+F%22">Sampaziotis F</searchLink>; Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK; Department of Medicine, University of Cambridge, Cambridge, UK; Centre for Translational Stem Cell Biology, Hong Kong, China. 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