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June 18, 2020

Quantitative Autofluorescence Following Gene Therapy With Voretigene Neparvovec

Author Affiliations
  • 1Department of Ophthalmology, Columbia University Irving Medical Center, New York, New York
  • 2Department of Ophthalmology, Hospital das Clínicas de Pernambuco, Empresa Brasileira de Serviços Hospitalares, Federal University of Pernambuco, Recife, Pernambuco, Brazil
  • 3Byers Eye Institute, Stanford University, Palo Alto, California
  • 4Department of Pathology and Cell Biology, Columbia University Medical Center, New York, New York
JAMA Ophthalmol. Published online June 18, 2020. doi:10.1001/jamaophthalmol.2020.2018

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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