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Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.

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作者单位: Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK. sahelja@upmc.edu py237@cam.ac.uk.;Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge CB2 0QQ, UK.;Moorfields Eye Hospital, London EC1V 2PD, UK.;UCL Institute of Ophthalmology, University College London, London EC1V 9EL, UK. [1] Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA. [2] IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.;Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy. [3] Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA. [4] Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA. [5] Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, 80336 Munich, Germany.;German Center for Neurodegenerative Diseases (DZNE), 80336 Munich, Germany.;Munich Cluster for Systems Neurology (SyNergy), 80336 Munich, Germany. [6] Department of Neuro-Ophthalmology and Emergencies, Rothschild Foundation Hospital, 75019 Paris, France.;Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France. [7] Department of Ophthalmology, University Hospital, LMU Munich, 80336 Munich, Germany. [8] Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.;Ottawa Hospital Research Institute and University of Ottawa Eye Institute, Ottawa, Ontario K1H 8L6, Canada. [9] GenSight Biologics, 75012 Paris, France. [10] GenSight Biologics, New York, NY 10016, USA. [11] Sorbonne Université, INSERM, CNRS, Institut de la Vision, 75012 Paris, France. [12] The Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, TN 37232, USA. [13] Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France. sahelja@upmc.edu py237@cam.ac.uk.;Sorbonne Université, INSERM, CNRS, Institut de la Vision, 75012 Paris, France.;Fondation Ophtalmologique A. de Rothschild, 25-29 Rue Manin, 75019 Paris, France.;Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA. [14]
DOI 10.1126/scitranslmed.aaz7423
PMID 33298565
发布时间 2025-02-11
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