Associated Papers
The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients.
Ashtari M et al.
Ophthalmology, 2017
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.
Bennett J et al.
Lancet (london, England), 2016
Long-term effect of gene therapy on Leber's congenital amaurosis.
Bainbridge JW et al.
The New England Journal of Medicine, 2015
Nature of the visual loss in observers with Leber's congenital amaurosis caused by specific mutations in RPE65.
Ripamonti C et al.
Investigative Ophthalmology & Visual Science, 2014
The human visual cortex responds to gene therapy-mediated recovery of retinal function.
Ashtari M et al.
The Journal of Clinical Investigation, 2011
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Maguire AM et al.
Lancet (london, England), 2009
Microarray-based mutation detection and phenotypic characterization of patients with Leber congenital amaurosis.
Yzer S et al.
Investigative Ophthalmology & Visual Science, 2006
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