1. Corey Haas, age 8, of Hadley, New York. LCA2 patient

2. What can we do about LCA2? What do we need to know first?

3. Dataset 1: A family history Genetic? Infectious? Environmental?

4. Dataset 2: Eye physiology Affected and unaffected individuals Clarity measurements Latency and amplitude of nerve transmission

5. Dataset 3: Molecular information Affected and unaffected individuals Measurements of rhodopsin, cis-retinal, trans-retinal, Na + concentration, mitochondria and DNA amounts Retinal

6. light Change in [Na + ] sends a signal down the optic nerve cis-retinal trans-retinal

7. light Change in [Na + ] sends a signal down the optic nerve RPE65

8. light Change in [Na + ] sends a signal down the optic nerve RPE65 LCA2!

9. light Change in [Na + ] sends a signal down the optic nerve RPE65 LCA2! Can we replace the defective RPE65 gene with a functional gene?

10. Corey Haas After RPE65 gene therapy on one eye