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. |
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| 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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| ISSN: | 2041-1723 |
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| DOI: | 10.1038/s41467-026-73631-x |
