Successful transduction of target gene mediated by adeno-associated virus 2 into lens epithelial cells in rats

Abstract

The Adeno-Associated Virus (AAV) has emerged as a promising candidate for delivery of genetic material, exhibiting significant potential in various clinical applications. Although multiple AAV serotypes have been shown to transduce ocular tissues, there have been few studies of AAV transduction of lens epithelial cells (LECs) in the ocular. In this study, we compared the efficiency of intravitreal injection of six AAV serotypes (AAV2, AAV5, AAV6, AAV8, AAV9, and AAVDJ) to transduce lens and retina in rats, The expression and localization of the reporter gene ZsGreen in the lens and retina were examined using immunofluorescence staining, and the relative expression of ZsGreen mRNA was detected using RT-qPCR. Our results demonstrated that AAV2 had the highest efficiency in transducing LECs. All six AAV serotypes could transduce the retina. To validate this observation, we further constructed an AAV2 vector with exogenous gene senescence marker protein 30 (SMP30) and performed intravitreal injection to successfully overexpress SMP30 in LECs of rats. our results provide a basis for the use of AAV vector-mediated gene therapy for lens diseases.