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 inflammatory disease has the potential for both therapeutic interventions and a method for studying mechanism of disease. An evolving philosophy on this subject would support the use of somatic gene therapy for ocular inflammatory disease, even if not life threatening. Major technical questions remain, including the use of the appropriate vector, the best methodology for the stable insertion into the genome, and the duration and intensity of expression of the transgene. Various transgenes encoding a wide variety of proteins can be envisaged for the insertion of genes. The study of gyrate atrophy, an hereditary ocular disorder and an excellent candidate for gene therapy, has given us enormous information 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 homologous recombination techniques for the development of animal models and later as a strategy for gene therapy. The use of gene therapy as a drug delivery system must also be considered. In addition, the elucidation of the various events controlling transcription for the expression of transgenes in various resident ocular cells is necessary.