ABSTRACT
Gene therapy holds great promise for the treatment of inherited disorders causing blindness. One such disorder is Leber congenital amaurosis (LCA), which accounts for 5% of all genetic retinal dystrophies. Success has been achieved in gene therapy treatment for the murine and canine models of LCA. The chapter discusses these model systems, the surgical techniques needed to deliver viral vectors carrying the correct gene to the subretinal space, and results to date. Several encouraging results have been achieved using delivery of RPE65 to homozygous mutant dogs via viral vectors. RPE65 gene into the subretinal space via subretinal injection. This ensures that the Retinal pigment epithelial cells, which normally express the RPE65 gene, will be exposed to the virus. The results from the human clinical trials for RPE65 –LCA will play an important role in dictating future directions/applications for gene therapy for retinal disease.
