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

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作者： Newman, Nancy J. ^[1] ; Yu-Wai-Man, Patrick ^[2] ; Carelli, Valerio ^[3] ; Moster, Mark L. ^[4] ; Biousse, Valerie ^[5] ; Vignal-Clermont, Catherine ^[6] ; Sergott, Robert C. ^[7] ; Klopstock, Thomas ^[8] ; Sadun, Alfredo A. ^[9] ; Barboni, Piero ^[10] ; DeBusk, Adam A. ^[11] ; Girmens, Jean Francois ; Rudolph, Guenther ; Karanjia, Rustum ; Taiel, Magali ; Blouin, Laure ; Smits, Gerard ; Katz, Barrett ; Sahel, Jose-Alain

作者单位： Ctr Hosp Natl Ophtalmol Quinze Vingts, Paris, France ^[1] Univ Cambridge, Cambridge Ctr Brain Repair, Cambridge, England ^[2] GenSight Biol, Paris, France ^[3] GenSight Biol, New York, NY USA ^[4] IRCCS Ist Sci Neurol Bologna, UOC Clin Neurol, Bologna, Italy ^[5] Wills Eye Hosp & Res Inst, Dept Neurol, Philadelphia, PA USA ^[6] Univ Calif Los Angeles, Sch Med, Doheny Eye Inst, Los Angeles, CA USA ^[7] Ludwig Maximilians Univ Munchen, Univ Hosp, Friedrich Baur Inst, Dept Neurol, Munich, Germany ^[8] Ludwig Maximilians Univ Munchen, Dept Ophthalmol, Univ Hosp, Munich, Germany ^[9] Emory Univ, Sch Med, Dept Ophthalmol, Atlanta, GA 30322 USA ^[10] Rothschild Fdn Hosp, Dept Neuroophthalmol & Emergencies, Paris, France ^[11]

关键词 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

发布时间 2021-07-09

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