Gene therapy has been a highly anticipated treatment for previously irreversible, blinding retinal disorders. In 2017, the US Food and Drug Administration approved the first gene therapy, voretigene neparvovec (VN), for treatment of retinal dystrophy caused by biallelic mutations in the RPE65 gene (OMIM 180069).1 Given the novel nature of gene therapy, it may be important to continue learning from this first group of treated patients. Herein, we present information gathered from the 6-year follow-up of a patient who was treated with VN.
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Levi SR, Oh JK, de Carvalho JRL, Mahajan VB, Tsang SH, Sparrow JR. Quantitative Autofluorescence Following Gene Therapy With Voretigene Neparvovec. JAMA Ophthalmol. Published online June 18, 2020. doi:10.1001/jamaophthalmol.2020.2018
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