Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer
Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Qu G, Zhou S, Zeiss C, Arruda VR, Acland GM, Dell'Osso LF, High KA, Maguire AM, Bennett J. Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer. Molecular Therapy 2008, 16: 458-465. PMID: 18209734, PMCID: PMC2842085, DOI: 10.1038/sj.mt.6300389.Peer-Reviewed Original ResearchConceptsLeber congenital amaurosisAnimal modelsCongenital amaurosisReversal of blindnessRetinal pigment epithelium cellsWeeks of injectionPigment epithelium cellsRPE65 formDose of vectorAppropriate target cellsVisual acuityVisual deficitsHistopathologic analysisAdeno-associated virusERG responsesRPE65 mutationsSubretinal deliveryEpithelium cellsMutant miceMinimal toxicityProtein expressionTarget cellsPupillary responseElectroretinogramAmaurosis