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Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.

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Title: Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Authors: Newman NJ; Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, USA., Yu-Wai-Man P; Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.; Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge, UK.; Moorfields Eye Hospital, London, UK.; UCL Institute of Ophthalmology, University College London, London, UK., Subramanian PS; Sue Anschutz-Rodgers University of Colorado Eye Center, University of Colorado School of Medicine, Aurora, CO, USA., Moster ML; Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA., Wang AG; Department of Ophthalmology, Taipei Veterans General Hospital, National Yang Ming Chiao Tung University, Taipei, Taiwan., Donahue SP; Department of Ophthalmology, Neurology, and Pediatrics, Vanderbilt University, and Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, TN, USA., Leroy BP; Department of Ophthalmology and Center for Medical Genetics, Ghent University Hospital, and Department of Head & Skin, Ghent University, Ghent, Belgium., Carelli V; IRCCS Istituto delle Scienze Neurologiche di Bologna, Programma di Neurogenetica, Bologna, Italy.; Unit of Neurology, Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Biousse V; Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, USA., Vignal-Clermont C; Department of Neuro Ophthalmology and Emergencies, Rothschild Foundation Hospital, Paris, France.; Department of Ophthalmology, Centre Hospitalier National D'Ophtalmologie des Quinze Vingts, Paris, France., Sergott RC; Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA., Sadun AA; Doheny Eye Institute, UCLA School of Medicine, Los Angeles, CA, USA., Rebolleda Fernández G; Department of Ophthalmology, Alcala University, Madrid, Spain., Chwalisz BK; Department of Ophthalmology, Massachusetts Eye & Ear, Harvard Medical School, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Banik R; Department of Ophthalmology, Icahn School of Medicine at Mount Sinai, New York, NY, USA., Bazin F; eXYSTAT, Data Management and Statistics, Paris, France., Roux M; GenSight Biologics, Paris, France., Cox ED; GenSight Biologics, Paris, France., Taiel M; GenSight Biologics, Paris, France., Sahel JA; Sorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France.; Fondation Ophtalmologique A. de Rothschild, Paris, France.; Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.; CHNO des Quinze-Vingts, Institut Hospitalo-Universitaire FOReSIGHT, INSERM-DGOS CIC, Paris, France.
Corporate Authors: LHON REFLECT Study Group
Source: Brain : a journal of neurology [Brain] 2023 Apr 19; Vol. 146 (4), pp. 1328-1341.
Publication Type: Randomized Controlled Trial; Clinical Trial, Phase III; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Oxford University Press Country of Publication: England NLM ID: 0372537 Publication Model: Print Cited Medium: Internet ISSN: 1460-2156 (Electronic) Linking ISSN: 00068950 NLM ISO Abbreviation: Brain Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
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  Data: <searchLink fieldCode="AU" term="%22Newman+NJ%22">Newman NJ</searchLink>; Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, USA.<br /><searchLink fieldCode="AU" term="%22Yu-Wai-Man+P%22">Yu-Wai-Man P</searchLink>; Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.; Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge, UK.; Moorfields Eye Hospital, London, UK.; UCL Institute of Ophthalmology, University College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Subramanian+PS%22">Subramanian PS</searchLink>; Sue Anschutz-Rodgers University of Colorado Eye Center, University of Colorado School of Medicine, Aurora, CO, USA.<br /><searchLink fieldCode="AU" term="%22Moster+ML%22">Moster ML</searchLink>; Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Wang+AG%22">Wang AG</searchLink>; Department of Ophthalmology, Taipei Veterans General Hospital, National Yang Ming Chiao Tung University, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Donahue+SP%22">Donahue SP</searchLink>; Department of Ophthalmology, Neurology, and Pediatrics, Vanderbilt University, and Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, TN, USA.<br /><searchLink fieldCode="AU" term="%22Leroy+BP%22">Leroy BP</searchLink>; Department of Ophthalmology and Center for Medical Genetics, Ghent University Hospital, and Department of Head & Skin, Ghent University, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22Carelli+V%22">Carelli V</searchLink>; IRCCS Istituto delle Scienze Neurologiche di Bologna, Programma di Neurogenetica, Bologna, Italy.; Unit of Neurology, Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.<br /><searchLink fieldCode="AU" term="%22Biousse+V%22">Biousse V</searchLink>; Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, USA.<br /><searchLink fieldCode="AU" term="%22Vignal-Clermont+C%22">Vignal-Clermont C</searchLink>; Department of Neuro Ophthalmology and Emergencies, Rothschild Foundation Hospital, Paris, France.; Department of Ophthalmology, Centre Hospitalier National D'Ophtalmologie des Quinze Vingts, Paris, France.<br /><searchLink fieldCode="AU" term="%22Sergott+RC%22">Sergott RC</searchLink>; Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA.<br /><searchLink fieldCode="AU" term="%22Sadun+AA%22">Sadun AA</searchLink>; Doheny Eye Institute, UCLA School of Medicine, Los Angeles, CA, USA.<br /><searchLink fieldCode="AU" term="%22Rebolleda+Fernández+G%22">Rebolleda Fernández G</searchLink>; Department of Ophthalmology, Alcala University, Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Chwalisz+BK%22">Chwalisz BK</searchLink>; Department of Ophthalmology, Massachusetts Eye & Ear, Harvard Medical School, Boston, MA, USA.; Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Banik+R%22">Banik R</searchLink>; Department of Ophthalmology, Icahn School of Medicine at Mount Sinai, New York, NY, USA.<br /><searchLink fieldCode="AU" term="%22Bazin+F%22">Bazin F</searchLink>; eXYSTAT, Data Management and Statistics, Paris, France.<br /><searchLink fieldCode="AU" term="%22Roux+M%22">Roux M</searchLink>; GenSight Biologics, Paris, France.<br /><searchLink fieldCode="AU" term="%22Cox+ED%22">Cox ED</searchLink>; GenSight Biologics, Paris, France.<br /><searchLink fieldCode="AU" term="%22Taiel+M%22">Taiel M</searchLink>; GenSight Biologics, Paris, France.<br /><searchLink fieldCode="AU" term="%22Sahel+JA%22">Sahel JA</searchLink>; Sorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France.; Fondation Ophtalmologique A. de Rothschild, Paris, France.; Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.; CHNO des Quinze-Vingts, Institut Hospitalo-Universitaire FOReSIGHT, INSERM-DGOS CIC, Paris, France.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Oxford+University+Press%22">Oxford University Press </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>0372537 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1460-2156 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200068950%22">00068950 </searchLink><i>NLM ISO Abbreviation: </i>Brain <i>Subsets: </i>MEDLINE
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        Value: 10.1093/brain/awac421
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