Purpose: Recently, we reported on novel hereditary dayblindness in sheep caused by a mutation in the CNGA3 gene (Reicher et al., Genomics 95:101-104, 2010). Since mutations in this gene can also cause achromatopsia in humans, we decided to use these sheep as a cone-enriched, large animal model to evaluate safety and efficacy of CNGA3 gene therapy.
Purpose: Recently, we reported on novel hereditary dayblindness in sheep caused by a mutation in the CNGA3 gene (Reicher et al., Genomics 95:101-104, 2010). Since mutations in this gene can also cause achromatopsia in humans, we decided to use these sheep as a cone-enriched, large animal model to evaluate safety and efficacy of CNGA3 gene therapy.