Abstract
Presentation Description :
Corneal scarring and neovascularization (NV) due to trauma, injury and/or infection are leading cause of global blindness. Tissue-targeted gene therapy is an effective and safe modality to treat corneal blindness with high bench-to-bedside translational potential for human application. The presentation will provide an overview of identified potent adeno-associated virus (AAV) and nanoparticle vectors, simple and minimally invasive vector delivery techniques for delivering genes into desired corneal cells for treating corneal disorders in vivo via an optimized customized single application topical method. Furthermore, presentation will discuss cornea-specific mechanisms driving pathologic process, novel molecular targets for interventional modalities, and single- and combination gene therapy approaches developed in our laboratory using established small and large animal in vivo and human in vitro and ex vivo models. The optimized gene therapy approaches could be easily applied in a clinical setting, if safety and toxicity are proven.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.