Ocular Gene Therapy Core

The Ocular Gene Therapy Core (OGTC) is an educational business activity operated as a cost-recovery core facility to produce research-grade adeno-associated virus (AAV) for both intramural and extramural investigators. The mission of this core is to develop and produce AAV vectors for gene-based discoveries and development of translational therapies to treat ocular diseases.


The cumulative resources of the OGTC are extensive. The OGTC has the capability of producing more than 2 million combinatorial AAV vector options from our in-house collection of more than 74 promoters, 357 gene cDNA’s, and 75 capsid serotypes and variants. Importantly, these AAV vectors have proven suitable for use in large animal models, including canine and primate models. Our collection of novel capsid variants is at the heart of the next generation of AAV vectors that will permit alternatives to subretinal delivery, and the transduction of difficult-to-target cell types. These are the vectors that will be at the cutting edge for broad spectrum treatments of inherited retinal disease and will move us beyond the state of the art single-gene approach.


Visit the Ocular Gene Therapy  Core site to learn more about using AAV vectors in your research.