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Leah Byrne; Gene delivery to the primate retina. Invest. Ophthalmol. Vis. Sci. 2019;60(9):4.
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Presentation Description :
Gene therapy is a highly promising approach for the treatment of inherited retinal disease. However, efficient and specific viral vector-mediated gene delivery to important cell types in the primate retina remains a major obstacle. Engineering of AAV vector capsids, through directed evolution and rational design, has resulted in more efficient vectors with shifted tropisms and enhanced ability to deliver therapeutic genes. We have created new AAV variants using high-throughput computationally guided screens performed in canines and in primates, and we describe new methods for creating vectors and promoters in the context of primate eyes. These new approaches for vector screening have allowed for the quantitative comparison of viruses, as well as the creation of an AAV atlas for optimal serotypes across cell types in the retina and brain.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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