Main Outcome Measures
Abbreviations and Acronyms:AAV2 (adeno-associated virus 2), D (diopters), FST (full-field stimulus threshold), LCA (Leber congenital amaurosis), RPE (retinal pigment epithelium), SD (standard deviation), VN (voretigene neparvovec-rzyl)
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Publication stageIn Press Journal Pre-Proof
This study was supported in part by National Institutes of Health /National Eye Institute Career Development Award K08EY030924 (AN), and a Research To Prevent Blindness Career Development Award (AN).
Financial Disclosure(s): The author(s) have made the following disclosure(s):
RAS: Consultant for Allergan, AGTC, EyePoint, Gyroscope, Leica, RegenXBio. CGB: Consultant for Janssen, research support from MeiraGTx, 4DMT, Spark Therapeutics. AMB: Consultant for DORC, Allergan, AGTC, ProQR, Occulus. AN: Consultant for Biogen, REGENXBIO, and Allergan Retina.
All authors have completed and submitted the ICMJE disclosures form.
Conception and design: Gange, Sisk, Besirli, Lee, Berrocal, Nagiel
Data collection: Gange, Sisk, Besirli, Havunjian, Schwartz, Borchert, Sengillo, Mendoza, Berrocal, Nagiel
Analysis and interpretation: Gange, Sisk, Besirli, Lee, Havunjian, Schwartz, Borchert, Sengillo, Mendoza, Berrocal, Nagiel
Obtained funding: Nagiel
Overall responsibility: Gange, Sisk, Besirli, Lee, Havunjian, Schwartz, Borchert, Sengillo, Mendoza, Berrocal, Nagiel
HUMAN SUBJECTS: Human subjects were included in this study. The human ethics committees at the Children’s Hospital Los Angeles, University of Miami, University of Cincinnati, and University of Michigan approved the study. All research adhered to the tenets of the Declaration of Helsinki. All participants provided informed consent.
No animal subjects were included in this study.