| Literature DB >> 22071974 |
C-M Lai1, M J Estcourt, R P Himbeck, S-Y Lee, I Yew-San Yeo, C Luu, B K Loh, M W Lee, A Barathi, J Villano, C-L Ang, R G van der Most, I J Constable, D Dismuke, R J Samulski, M A Degli-Esposti, E P Rakoczy.
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 not elicit cell-mediated immunity. Biodistribution analysis showed that AAV2.sFlt-1 could be detected only in the eye and not in the other organs tested. These data indicate that gene therapy with subretinal AAV2.sFlt-1 is safe and well tolerated, and therefore promising for the long-term treatment of neovascular diseases of the eye.Entities:
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Year: 2011 PMID： 22071974 DOI： 10.1038/gt.2011.169
Source DB: PubMed Journal: Gene Ther ISSN： 0969-7128 Impact factor: 5.250