The Byrne Lab develops gene therapies for retinal disease, including a diverse group of blinding disorders that have a profound impact on the quality of life of patients. There are currently no effective treatments for most forms of inherited retinal degeneration. However, gene therapy, in which therapeutic genetic material is transferred to cells, is a highly promising approach to treating retinal disease. The Byrne lab is exploring gene augmentation, optogenetics, and genome editing approaches to treat inherited and age-related forms of blindness.

The most effective approach to deliver therapeutic genes to cells is using viruses, which have evolved over millions of years as highly efficient gene delivery systems. In viral vector-mediated gene therapy, the viral genome is replaced with a cassette containing a promoter driving expression of a therapeutic transgene. The virus is then injected into the tissue, where it infects cells, carrying its genetic payload to the nucleus, and resulting in expression of a therapeutic protein. However, the viruses found in nature are not naturally suited to deliver gene therapies to retinal cells. The Byrne Lab uses naturally occurring viruses as starting material, and implements high throughput, computationally guided and structurally informed approaches to engineer viral vectors with new abilities to deliver therapies to cells in the eye.

Our group is working to create a publicly available online database of AAV tropism in the primate eye. We are leveraging our scAAVengr AAV engineering platform to determine the number and types of cells targeted by the most widely used and promising engineered AAV serotypes at clinically relevant titers. This database will provide quantitative information, with single-cell resolution, about the level of transgene expression achieved, in order to guide future clinical trials. Watch this space for further information.