Next-Generation AAV Delivery Systems for In Vivo Gene Therapy

Our laboratory develops next-generation AAV-mediated gene delivery systems through an integrated framework that combines directed evolution, rational vector design, and biomaterial-mediated delivery strategies. This approach enables the engineering of highly cell- and tissue-specific AAV vectors, receptor–ligand–guided targeting systems, and biomaterial-enabled platforms for localized, controlled, and efficient in vivo gene delivery.
We focus on high-precision in vivo targeting across a broad range of biologically and clinically challenging tissues, including the retina, inner ear (hearing loss), blood–brain barrier, central nervous system (neurons, astrocytes, microglia, and oligodendrocytes), neuromuscular systems, immune cells (T, B, NK cells, and macrophages), cardiovascular tissues, respiratory epithelia, and diverse cancer cell types. These efforts aim to enable targeted genetic interventions across a broad spectrum of inherited and acquired diseases.
To advance beyond vector discovery toward clinically relevant therapies, our research integrates immune-evasive AAV engineering, scalable vector manufacturing and purification, and large-animal validation, including studies in non-human primate models. In parallel, we collaborate closely with clinicians, academic investigators, and industry partners to translate these technologies into clinically robust and commercially viable gene therapy products.