of the Retina in the 4 Years Following rAAV-mediated Gene Transfer in the RPE65 Null Mutation Dog
Retinal gene transfer studies performed in our laboratories in groups of 4–42 monthold RPE65 null mutation dogs have shown remarkable functional improvement following a single unilateral subretinal injection of a recombinant adeno-associated virus vector, serotype 2, rAAV2/2, cDNA for dog RPE65 with a cytomegalo virus (CMV) promoter (Narfström et al., 2003a,b, 2005; Ford et al., 2003). The purpose of the present investigation was to further assess long-term therapeutic effects of the gene transfer performed in a group of young RPE65 null mutation dogs and to correlate the functional effects of therapy to morphologic findings up to 4 years following the gene transfer. In addition, retinas of affected dogs were examined for histologic correlates of spots and color changes seen by ophthalmoscopy in both treated and untreated affected dogs.
KeywordsGene Transfer Outer Nuclear Layer Photoreceptor Outer Segment Severe Degenerative Change Childhood Blindness
Unable to display preview. Download preview PDF.
- Le Meur, G., Stieger, K., Weber, M., Deschamps, J.Y., et al., 2006,Restoration of vision in RPE65-deficient Briard dogs using an AAV-serotype 4 vector that specifically targets the retinal pigment epithelium, Gene Therapy online, doi:10.1038/sj.gt. 3302861.Google Scholar
- Narfströ m, K., Katz, M.L., Bragadottir, R., Seeliger, M., Boulanger, A., Redmoond, R.M., Caro, L., Lai, C-M., Rakoczy, P.E., 2003a, Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog, Invest Ophthalmol Vis Sci, 44: 1663–1672.Google Scholar
- Narfströ m, K., Katz, M.L., Bragadottir, R., et al., 2003b, In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement, J Hered, 94: 31–37.Google Scholar