Gene therapies have recently emerged as promising strategies for treatment of previously incurable or poorly managed diseases. These hopes are particularly high in ophthalmology, as the eye is considered exceptionally suited for gene therapy. Expansion of gene therapy products may change the clinical course of treatment and give new chances to many patients. In this review, we address treatment possibilities and delivery methods as well as challenges and uncertainties related to gene therapy. We present inherited and acquired diseases which are subject to studies within this area, summarize current trends in ocular gene therapy, and indicate the future directions.