ADENO ASSOCIATED VIRAL VECTORS FOR TARGETED TRANSDUCTION OF RETINAL PIGMENT EPITHETIAL CELLS

摘要

A method for specifically targeting a gene product to a retinal pigment epithelial cell is provided, along with adeno-associated viruses (AAVs) useful therein. The method involves delivering to a subject's eye a dose of about 109 genome copies of an AAV7 viral vector and a minigene comprising sequences encoding the gene product targeted to the RPE cells. A synthetic RPE gene 65 is also described. Suitably, this synthetic RPE gene is under the control of regulatory control sequences which direct expression of the RPE65 protein in RPE cells and is delivered via the AAV7 viral vector.