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
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..View full abstract
This work was supported by the Juvenile Diabetes Research Foundation (USA), the National Institutes of Health (USA), the National Health and Medical Research Council (Australia) and Westpac grant-in-aid (Australia). MDE holds a NHMRC Principal Research Fellowship.