Abstract: Diabetic retinopathy (DR), one of the common complications of diabetes, is the main cause of blindness. Due to the limitations of the existing clinical treatment methods, it is urgent to develop new targets or/and new therapeutic drugs. This review summarizes the clinical trials of anti-DR drugs in recent years, and we note that gene therapy is a potential direction for DR treatment development. Due to the characteristics of ocular structure, including small size, a relatively independent organ, immune privilege and the opportunity for local administration, gene therapy could well be advantageous in the treatment of DR. Furthermore, the long-term therapeutic effects of gene therapy also improve compliance by DR patients. All these indicate that gene therapy is likely a future direction for development of DR therapies.