A drug delivery system of <i>HIF-1α</i> siRNA nanoparticles loaded by mesenchymal stem cells on choroidal neovascularization

<b>Aim:</b> To assess mesenchymal stem cells (MSCs) as carriers for <i>HIF-1α</i> siRNA-loaded nanoparticles (NPs) for targeted therapy of experimental choroidal neovascularization (CNV).<b>Materials & methods:</b> A poly (lactic-co-glycolic acid) (PLGA)-core/lipid-shell hybrid NP was designed. The transfection efficacy of MSCs with the hybrid NPs was assessed. Mice were intravenously injected with MSCs after laser photocoagulation and CNV was assessed at 7 days post-injection.<b>Results & conclusion:</b> The transfection efficiency of hybrid NPs into MSCs was 72.7%. <i>HIF-1α</i> mRNA expression in 661w cells co-cultured with MSC-hybrid-siRNA NPs was significantly lower. Intravenous delivery of MSC-hybrid-siRNA NPs greatly reduced CNV area and length. Intravenous injection of MSC-hybrid-siRNA NPs achieved therapeutic efficacy in reducing CNV area. The MSC-mediated homing enabled targeted inhibition of ocular angiogenesis.