2006
Safety in Nonhuman Primates of Ocular AAV2-RPE65, a Candidate Treatment for Blindness in Leber Congenital Amaurosis
Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW. Safety in Nonhuman Primates of Ocular AAV2-RPE65, a Candidate Treatment for Blindness in Leber Congenital Amaurosis. Human Gene Therapy 2006, 17: 845-858. PMID: 16942444, DOI: 10.1089/hum.2006.17.845.Peer-Reviewed Original ResearchConceptsLeber congenital amaurosisOptic nerveGood Laboratory Practice safety studiesCongenital amaurosisObserved adverse effect levelSafety studiesRPE65-mutant dogsSingle intraocular injectionVector-injected eyesHeterogeneous disease groupNormal cynomolgus monkeysAdverse effect levelFuture human trialsGene transfer trialsDose-dependent mannerHigh vector dosesPresurgical recordingsRetinal histopathologyInjected eyeControl eyesIntraocular injectionRetinal injuryCentral retinaThickness abnormalitiesClinical examination
2005
CNTF induces dose-dependent alterations in retinal morphology in normal and rcd-1 canine retina
Zeiss CJ, Allore HG, Towle V, Tao W. CNTF induces dose-dependent alterations in retinal morphology in normal and rcd-1 canine retina. Experimental Eye Research 2005, 82: 395-404. PMID: 16143329, DOI: 10.1016/j.exer.2005.07.014.Peer-Reviewed Original ResearchConceptsCiliary neurotrophic factorInner retinal thicknessRetinitis pigmentosaRetinal thicknessGanglion cellsEffects of CNTFInner nuclear layer thicknessNormal retinal electrophysiologyGanglion cell morphologyOuter nuclear layerWild-type retinasDose-dependent mannerDose-dependent alterationsContralateral eyeControl dogsNeurotrophic factorExpression of rhodopsinRetinal electrophysiologyNuclear layerProtective effectRetinal morphologyEntire retinaAnimal modelsCone arrestinCanine model