Full-field Scotopic Threshold Improvement after Voretigene Neparvovec-rzyl Treatment Correlates with Chorioretinal Atrophy.

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作者： Katarina,Stingl ^[1] ; Krunoslav,Stingl ^[1] ; Hillary,Schwartz ^[2] ; Mark W,Reid ^[2] ; Melanie,Kempf ^[1] ; Spyridon,Dimopoulos ^[3] ; Friederike,Kortuem ^[3] ; Mark S,Borchert ^[4] ; Thomas C,Lee ^[4] ; Aaron,Nagiel ^[5]

作者单位： University Eye Hospital, Center for Ophthalmology, University of Tübingen, Tübingen, Germany; Center for Rare Eye Diseases, University of Tübingen, Tübingen, Germany. ^[1] The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California. ^[2] University Eye Hospital, Center for Ophthalmology, University of Tübingen, Tübingen, Germany. ^[3] The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California; Roski Eye Institute, Department of Ophthalmology, Keck School of Medicine, University of Southern California, Los Angeles, California. ^[4] The Vision Center, Department of Surgery, Children's Hospital Los Angeles, Los Angeles, California; Roski Eye Institute, Department of Ophthalmology, Keck School of Medicine, University of Southern California, Los Angeles, California. Electronic address: anagiel@chla.usc.edu. ^[5]

关键词 Chorioretinal atrophy FST Gene therapy Inherited retinal dystrophy