2017
Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs
Ye GJ, Komáromy AM, Zeiss C, Calcedo R, Harman CD, Koehl KL, Stewart GA, Iwabe S, Chiodo VA, Hauswirth WW, Aguirre GD, Chulay JD. Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs. Human Gene Therapy 2017, 28: 197-207. PMID: 29020838, PMCID: PMC5733651, DOI: 10.1089/humc.2017.125.Peer-Reviewed Original ResearchConceptsHuman CNGB3Immune responseHigh dosesT cell immune responsesHigh-dose groupLow-dose groupInherited retinal disorderImmune-mediated toxicitiesHigher vector doseCone photoreceptor functionDifferent dose levelsFocal chorioretinitisRetinal toxicityVisual acuityInflammatory responseAAV vectorsRetinal disordersSubretinal injectionCone functionDose levelsCone photoreceptorsPhotoreceptor functionCNGB3AAV5 vectorVector dose
2008
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