Journal article

Preclinical safety evaluation of subretinal AAV2.sFlt-1 in non-human primates

C-M Lai, MJ Estcourt, RP Himbeck, S-Y Lee, I Yew-San Yeo, C Luu, BK Loh, MW Lee, A Barathi, J Villano, C-L Ang, RG van der Most, IJ Constable, D Dismuke, RJ Samulski, MA Degli-Esposti, EP Rakoczy

GENE THERAPY | NATURE PUBLISHING GROUP | Published : 2012

Abstract

We report on the long-term safety of AAV2.sFlt-1 (a recombinant adeno-associated virus serotype 2 carrying the soluble form of the Flt-1 receptor) injection into the subretinal space of non-human primates. Levels of sFlt-1 protein were significantly higher (P<0.05) in the vitreous of four out of five AAV2.sFlt-1-injected eyes. There was no evidence of damage to the eyes of animals that received subretinal injections of AAV2.sFlt-1; ocular examination showed no anterior chamber flare, normal fundus and electroretinography responses equivalent to those observed before treatment. Notably, immunological analysis demonstrated that gene therapy involving subretinal injection of AAV2.sFlt-1 does no..

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University of Melbourne Researchers