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Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models.

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Title: Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models.
Authors: Cozmescu CA; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK. claudiu.cozmescu.15@ucl.ac.uk.; Great Ormond Street Institute of Child Health, University College London, London, UK. claudiu.cozmescu.15@ucl.ac.uk., Nazari M; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK., Touramanidou L; Great Ormond Street Institute of Child Health, University College London, London, UK., Gurung S; Great Ormond Street Institute of Child Health, University College London, London, UK., Perocheau D; Great Ormond Street Institute of Child Health, University College London, London, UK., Sebire N; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK., Hu YT; Great Ormond Street Institute of Child Health, University College London, London, UK., Goldsworthy S; Great Ormond Street Institute of Child Health, University College London, London, UK., Burden JJ; Laboratory for Molecular Cell Biology, University College London, London, UK., Khalil Y; Great Ormond Street Institute of Child Health, University College London, London, UK., Doykov I; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK., Counsell JR; Division of Surgery and Interventional Science, University College London; Royal Free Hospital, 9Th Floor (East), London, UK., Karda R; EGA Institute for Women's Health, University College London, London, UK., Castellano S; Great Ormond Street Institute of Child Health, University College London, London, UK., Mills P; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK., Clayton P; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK., Heywood W; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK., Moulding D; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK., Waddington SN; EGA Institute for Women's Health, University College London, London, UK., Baruteau J; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.; Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust; Great Ormond Street, London, UK., Turchiano G; Great Ormond Street Institute of Child Health, University College London, London, UK., Gissen P; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK. p.gissen@ucl.ac.uk.; Great Ormond Street Institute of Child Health, University College London, London, UK. p.gissen@ucl.ac.uk.; Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust; Great Ormond Street, London, UK. p.gissen@ucl.ac.uk.
Source: Nature communications [Nat Commun] 2026 Jun 19; Vol. 17 (1). Date of Electronic Publication: 2026 Jun 19.
Publication Type: Journal Article
Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Cozmescu+CA%22">Cozmescu CA</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK. claudiu.cozmescu.15@ucl.ac.uk.; Great Ormond Street Institute of Child Health, University College London, London, UK. claudiu.cozmescu.15@ucl.ac.uk.<br /><searchLink fieldCode="AU" term="%22Nazari+M%22">Nazari M</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Touramanidou+L%22">Touramanidou L</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Gurung+S%22">Gurung S</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Perocheau+D%22">Perocheau D</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Sebire+N%22">Sebire N</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.<br /><searchLink fieldCode="AU" term="%22Hu+YT%22">Hu YT</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Goldsworthy+S%22">Goldsworthy S</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Burden+JJ%22">Burden JJ</searchLink>; Laboratory for Molecular Cell Biology, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Khalil+Y%22">Khalil Y</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Doykov+I%22">Doykov I</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Counsell+JR%22">Counsell JR</searchLink>; Division of Surgery and Interventional Science, University College London; Royal Free Hospital, 9Th Floor (East), London, UK.<br /><searchLink fieldCode="AU" term="%22Karda+R%22">Karda R</searchLink>; EGA Institute for Women's Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Castellano+S%22">Castellano S</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Mills+P%22">Mills P</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Clayton+P%22">Clayton P</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Heywood+W%22">Heywood W</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Moulding+D%22">Moulding D</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Waddington+SN%22">Waddington SN</searchLink>; EGA Institute for Women's Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Baruteau+J%22">Baruteau J</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.; Great Ormond Street Institute of Child Health, University College London, London, UK.; Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust; Great Ormond Street, London, UK.<br /><searchLink fieldCode="AU" term="%22Turchiano+G%22">Turchiano G</searchLink>; Great Ormond Street Institute of Child Health, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Gissen+P%22">Gissen P</searchLink>; NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK. p.gissen@ucl.ac.uk.; Great Ormond Street Institute of Child Health, University College London, London, UK. p.gissen@ucl.ac.uk.; Metabolic Medicine, Great Ormond Street Hospital for Children NHS Foundation Trust; Great Ormond Street, London, UK. p.gissen@ucl.ac.uk.
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