Date of this Version
Proceedings of the National Academy of Sciences
We appreciate the interest shown by TownesAnderson in our article examining the natural history of retinal degeneration in Leber congenital amaurosis caused by retinal pigment epithelium-specific protein 65kDa (RPE65) mutations and evaluating the consequences of gene augmentation therapy. Townes-Anderson’s remarks focused on the final phrase of the last sentence of the Discussion of our article. In the full sentence, we suggested that in the future, agents to reduce cell death could be delivered in combination with a more advanced version of the gene augmentation therapy that reaches not only remaining rods and extrafoveal cones but also foveal cone photoreceptors.
© 2013 National Academy of Sciences.
Cideciyan, A. V., Jacobson, S. G., Beltran, W. A., Hauswirth, W. W., & Aguirre, G. D. (2013). Reply to Townes-Anderson: RPE65 Gene Therapy Does Not Alter the Natural History of Retinal Degeneration. Proceedings of the National Academy of Sciences, 110 (19), E1706-. http://dx.doi.org/10.1073/pnas.1304296110
Date Posted: 20 April 2018
This document has been peer reviewed.