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Correcting the Defects: Gene Therapy for Retinal Degeneration
1Bennett J., 1Dejneka N., 1Jacobson S., 1Aleman T., 1Maguire A., 2Acland G., 2Aguirre G.,
1University of Pennsylvania, Scheie Eye Institute, 310 Stellar-Chance Labs (Philadelphia)
2Cornell University (Ithaca)
Purpose: Recent success in delivering vision to a canine model of a severe, early onset blinding disease, Leber congenital Amaurosis (LCA) (Acland et al, 2001, Nature Genetics 28:92) invites speculation that the human disease could be treated similarly. In order to further characterize the therapeutic effects of such treatments and also to identify the limitations and potential complications of such treatments, we have compared the effects of delivery of the RPE65-encoding cDNA in mice and dogs lacking the RPE65 protein.
Methods: We evaluated the effects of age of treatment, dose of therapeutic material, target area of the retina, species of origin of the cDNA, cellular specificity of the treatment, and previous immunological history of the subjects. We also compared the utility of several different viral vectors (including adeno-associated viruses (AAVs) of different serotypes and lentivirus) in administering the treatment.
Results: Therapeutic effects were found using vectors which resulted in efficient delivery of the RPE65 to retinal cells. Therapeutic effects were identified at a range of different ages and these effects were quite stable. The most common complications of treatment occurred within the early postoperative period and included inflammatory changes due to virus/transgene delivery. These complications could be managed effectively, however, with local steroid administration.
Conclusions: The potential benefits of gene therapy for LCA appear to outweigh the risks in both mice and dogs. This bodes well for eventual human application to this and other retinal degenerative diseases.