Gene Therapy using SiRNA for Treatment of Ocular Neovascularization

Abstract

Recently, gene therapy has become a novel strategy for severe disease treatment, especially using small interfering RNA (siRNA). SiRNA is about 20-25 nucleotide-long, generated from long double-strand RNA (dsRNA) after the cleavage of DICER, to target and degrade complementary mRNA through the RNA interference (RNAi) pathway. The targeted delivery of siRNA has become an important gene silencing strategy because of its highly potent down-regulation of gene expression in targeting cells. Currently, siRNA is being examined as a potential therapeutic treatment to reduce ocular neovascularization. However, successful gene therapy depends on an efficient delivery system. Cationic liposomes are composed of positively charged lipid, these liposomes can condense nucleic acid into small particles and protect loaded genes from nuclease degradation. Therefore, cationic liposome is considered a good candidate for gene delivery. This review will discuss the development of gene therapy for ocular neovascularization. In addition, current delivery systems of cationic liposome with PEGylation on lipid are also introduced.