Gene Delivery

Project Summary Gene Delivery Core Vision research has benefited enormously by the advent of gene delivery technologies, aimed at understanding the development, physiology, and pathology of the visual system, and in developing strategies for treating ocular disease. Especially important have been advances in AAV based gene therapy, directed evolution of new AAV capsid variants, restoration of retinal responses in degenerative diseases by optogenetic proteins, and CRISPR/Cas9 based gene editing technology for editing endogenous genes in the eye and the brain. Over the past two and half decades, the Gene Delivery Core (GDM) has played an essential role in helping our Vision Science researchers employ these technologies, fostering collaboration among labs, and attracting new labs into pursuing questions that are relevant for vision science. Our goal in the next funding period is to continue providing this essential service to Core investigators, their labs, and the larger Vision Science community at UC Berkeley. The aims of the Core are 1) Design and produce viral vectors for Vision Science researchers, 2) Introduce new gene delivery technologies to Vision Science researchers, 3) Assist UC Berkeley Vision Science researchers in generating transgenic animal models.