Gene therapy prospects in ophthalmology

PURPOSE: Presentation of newest achievements from borderland of ophthalmology and molecular biology and their clinical employment areas in the therapy of eye diseases. MATERIAL AND METHODS: MEDLINE database has been searched for terms gene, gene therapy, ocular, eye in title and summary fields. RESULTS: Based on the latest literature eye disorders have been selected that are currently in focus of gene therapy. Experimental approaches in vitro as well as in vivo have been reviewed. CONCLUSIONS: Modern ophthalmology profits more and more from newest achievements in molecular biology such as gene therapy. Transfer of additional genes to selected target cells within eye is able to change function and allows to achieve required effects. It is performed through employment of so called vectors, small particles allowing the gene to be transferred. Presently most often used vectors are derived from viruses (retrovirus, adenovirus, viruses AAV). Experimental studies have been performed in following eye diseases: retinitis pigmentosa, Leber congenital amaurosis, glaucoma, retinoblastoma, postoperative posterior capsule opacification, corneal diseases, age related macular degeneration, uveal melanoma. In each case the experimental results were promising. However, possible side effects of such therapy are not sufficiently known. There have been no clinical gene therapy studies in ophthalmology so far.