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Dr. Bennett and her team at Penn’s Center for Advanced Retinal and Ocular Therapeutics (CAROT) have developed an adeno associated viral vector for the delivery of an optimized CNGA3 gene. Mutations in the alpha subunit of the cone photoreceptor cyclic nucleotide-gated channel (CNGA3) genes underlie ~25% of Achromatopsia cases.
When delivered to the retina of the CNGA3-/- mice, these constructs have shown a dose dependent expression in the targeted cone cells with minimal adverse effects on the rod function. Similar results in humans would offer a potential cure for Achromatopsia.