PERSPECTIVES ON GENE-THERAPY IN THE TREATMENT OF OCULAR INFLAMMATION

Gene therapy may become a powerful therapeutic strategy. However, the application of this method in the treatment of ocular disease presents us with interesting and unique questions. Gene therapy for ocular inf lammatory disease has the potential for both therapeutic interventions and a method for studying mechanisms of disease. An evolving philosop hy on this subject would support the use of somatic gene therapy for o cular inflammatory disease, even if not life threatening. Major techni cal questions remain, including the use of the appropriate vector, the best methodology for the stable insertion into the genome, and the du ration and intensity of expression of the transgene. Various transgene s encoding a wide variety of proteins can be envisaged for the inserti on of genes. The study of gyrate atrophy, an hereditary ocular disorde r and an excellent candidate for gene therapy, has given us enormous i nformation in the development of practical therapeutic strategies, as have in vitro studies of gene insertion. Future concerns will need to concentrate on the use of better methods for gene insertion and homolo gous recombination techniques for the development of animal models and later as a strategy for gene therapy. The use of gene therapy as a dr ug delivery system must also be considered. In addition, the elucidati on of the various events controlling transcription for the expression of transgenes in various resident ocular cells is necessary.