Grounded in a thorough understanding of the basic biology of adeno-associated viruses, our discovery research aims to develop improved gene transfer vehicles for a variety of targets.
Our Discovery Research Group is focused on understanding and improving AAV-based gene therapy and gene editing technologies.
Capsids: We continue the groundbreaking work of isolating new families of AAVs from nature and characterizing their gene-transfer potential. In addition to this foundational work, we employ all the tools of modern protein engineering to tailor natural AAV capsids to specific gene therapy and gene editing applications.
Transgenes: We use deep mechanistic study of AAV function, safety, and episomal regulation in vivo to inform the optimization of gene therapy expression cassettes.
Gene editing: We are improving the safety and efficacy of gene editing and gene editing delivery technologies. This includes technology development to measure and modulate on-target and off-target editing activities in vivo.
Infectious Disease: We continue the essential work to develop efficient AAV-based treatments, prophylaxis, and vaccines to respiratory viruses.