Expression of the green fluorescent reporter protein gfp exclusively in a blue wavelength sensitive cone in monkey retina.

We are engineering adeno associated viruses (AAV) to target specific cell types in the retina. We have developed cell-specific AAV vectors can transfer gene constructs specifically to rods, cones, M�ller glia or RPE cells in the eye. This figure shows expression of the green fluorescent reporter protein gfp exclusively in a blue?wavelength sensitive cone in monkey retina. We plan to use these targeted vectors for gene replacement therapy for recessive eye diseases. Genetically dominant eye diseases are gain-of-function mutations and cannot be corrected by adding a corrective gene. For these studies, mutation-specific, therapeutic strategies are being developed to 'knock-down' the mRNA from the mutant allele by expression of ribozymes in AAV.