Introduction: Overexpression of VEGF has been identified to be associated with many pathologic processes such as tumors and retinopathy. Inhibiting uncontrolled growth of VEGF is a promising strategy to treat these diseases. Currently small molecule inhibitors and monoclonal antibodies are the primary treatment. However, complex development, short half-life, limited effectiveness and potential systemic side effects limited their applications. Highly effective and safe therapeutic technologies are highly desirable to meet the growing clinical needs. RNA interference (RNAi) technology, inhibits special gene activity at the post transcriptional level and reduces the expression of relevant proteins, holding great potential due to its easy design and high efficacy. Some molecules based on RNAi have been investigating in different clinical trials.
Areas covered: In this article, we review and consider current advances in the application of RNAi technology and potential future clinical strategies.
Expert opinion: RNAi technology has a promising future in anti-VEGF treatment, most of the investigations are encouraging and exciting. More anti-VEGF candidates will enter clinical trials and may be a novel therapeutic strategy.
Keywords: RNA interfering; VEGF; age-related macular degeneration; angiogenesis; anti-VEGF; gene delivery; small interfering RNA; tumor.