Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset期刊界 All Journals 搜尽天下杂志传播学术成果专业期刊搜索期刊信息化学术搜索

Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset

Authors:	Nancy J. Newman Patrick Yu-Wai-Man Valerio Carelli Mark L. Moster Valerie Biousse Catherine Vignal-Clermont Robert C. Sergott Thomas Klopstock Alfredo A. Sadun Piero Barboni Adam A. DeBusk Jean François Girmens Günther Rudolph Rustum Karanjia Magali Taiel Laure Blouin Gerard Smits Barrett Katz Maria Massini

Affiliation:	23. Centre Hospitalier National d’Ophtalmologie des Quinze Vingts, Paris, France and Department of Neuro Ophthalmology and Emergencies, Rothschild Foundation Hospital, Paris, France;24. Department of Neurology, Friedrich-Baur-Institute, and Department of Ophthalmology, University Hospital, Ludwig-Maximilians-University Munich, TUMCells Interdisciplinary Center for Cellular Therapies, TUM School of Medicine, Munich, Germany;25. Doheny Eye Institute / UCLA School of Medicine Los Angeles, California;26. Emory University School of Medicine, Atlanta, Georgia;27. IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Bologna, Italy, and Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy;28. Moorfields Eye Hospital, London, UK and UCL Institute of Ophthalmology, University College, London, London, UK;29. Wills Eye Hospital and Sidney Kimmel Medical College of Thomas Jefferson University, Philadelphia, Pennsylvania;1. Departments of Ophthalmology, Neurology, and Neurological Surgery, Emory University School of Medicine, Atlanta, Georgia;2. Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom;3. Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals, Cambridge, United Kingdom;4. Moorfields Eye Hospital, London, United Kingdom;5. UCL Institute of Ophthalmology, University College London, London, United Kingdom;6. IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Bologna, Italy;7. Unit of Neurology, Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy;8. Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, Pennsylvania;9. Department of Neuro-Ophthalmology and Emergencies, Rothschild Foundation Hospital, Paris, France;10. Centre Hospitalier National d’Ophtalmologie des Quinze Vingts, Paris, France;11. Department of Neurology, Friedrich-Baur-Institute, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany;12. German Center for Neurodegenerative Diseases, Munich, Germany;13. Munich Cluster for Systems Neurology (SyNergy), Munich, Germany;14. Doheny Eye Institute/UCLA School of Medicine, Los Angeles, California;15. Department of Ophthalmology, University Hospital, LMU Munich, Munich, Germany;16. Department of Ophthalmology, University of Ottawa Eye, Ottawa, Ontario, Canada;17. GenSight Biologics, Paris, France;18. GenSight Biologics, New York, New York;19. Sorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France;20. Fondation Ophtalmologique A. de Rothschild, Paris, France;21. Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania;22. CHNO des Quinze-Vingts, Institut Hospitalo-Universitaire FOReSIGHT, INSERM-DGOS CIC, Paris, France

Abstract:

Keywords:	Leber Hereditary Optic Neuropathy intravitreal gene therapy efficacy safety bilateral visual improvement best-corrected visual acuity Phase 3 randomized double-masked clinical trial contrast sensitivity Humphrey visual field perimetry retinal anatomic measures Quality of life BCVA" },{" #name" :" keyword" ," $" :{" id" :" kwrd0070" }," $$" :[{" #name" :" text" ," _" :" best-corrected visual acuity cDNA" },{" #name" :" keyword" ," $" :{" id" :" kwrd0080ww" }," $$" :[{" #name" :" text" ," _" :" complementary DNA CRR" },{" #name" :" keyword" ," $" :{" id" :" kwrd0080" }," $$" :[{" #name" :" text" ," _" :" clinically relevant recovery CS" },{" #name" :" keyword" ," $" :{" id" :" kwrd0090" }," $$" :[{" #name" :" text" ," _" :" contrast sensitivity dB" },{" #name" :" keyword" ," $" :{" id" :" kwrd0100" }," $$" :[{" #name" :" text" ," _" :" decibels EMA" },{" #name" :" keyword" ," $" :{" id" :" kwrd0110" }," $$" :[{" #name" :" text" ," _" :" European Medicines Agency ETDRS" },{" #name" :" keyword" ," $" :{" id" :" kwrd0120" }," $$" :[{" #name" :" text" ," _" :" Early Treatment Diabetic Retinopathy Study HVF" },{" #name" :" keyword" ," $" :{" id" :" kwrd0130" }," $$" :[{" #name" :" text" ," _" :" Humphrey visual field ITT" },{" #name" :" keyword" ," $" :{" id" :" kwrd0140" }," $$" :[{" #name" :" text" ," _" :" intention-to-treat IVT" },{" #name" :" keyword" ," $" :{" id" :" kwrd0140rr" }," $$" :[{" #name" :" text" ," _" :" intravitreal injection LHON" },{" #name" :" keyword" ," $" :{" id" :" kwrd0150" }," $$" :[{" #name" :" text" ," _" :" Leber hereditary optic neuropathy LogCS" },{" #name" :" keyword" ," $" :{" id" :" kwrd0160" }," $$" :[{" #name" :" text" ," _" :" logarithm of contrast sensitivity logMAR" },{" #name" :" keyword" ," $" :{" id" :" kwrd0170" }," $$" :[{" #name" :" text" ," _" :" logarithm of the minimal angle of resolution mtDNA" },{" #name" :" keyword" ," $" :{" id" :" kwrd0180" }," $$" :[{" #name" :" text" ," _" :" mitochondrial DNA MTS" },{" #name" :" keyword" ," $" :{" id" :" kwrd0190" }," $$" :[{" #name" :" text" ," _" :" mitochondrial targeting sequence RGC" },{" #name" :" keyword" ," $" :{" id" :" kwrd0200" }," $$" :[{" #name" :" text" ," _" :" retinal ganglion cell RNFL" },{" #name" :" keyword" ," $" :{" id" :" kwrd0210" }," $$" :[{" #name" :" text" ," _" :" retinal nerve fiber layer
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