#457 Ocular Toxicity and Efficacy of rAAV2tYF-PR1.7 -hCNGB3 Vector Following Subretinal Injection in a Mouse Model of Achromatopsia

Mutations in the cone-specific cyclic nucleotide-gated channel beta subunit (CNGB3) gene account for about 50% of cases of achromatopsia, an autosomal recessive retinal disease affecting functionality of cone photoreceptors1. The phenotype of CNGB3-/mice is similar to that in humans, specifically loss of cone function and early onset, slowly progressive cone degeneration, making it an appropriate disease model to study both toxicity and efficacy of the treatment2,3,.