Document Type

Journal Article

Date of this Version

5-7-2013

Publication Source

Proceedings of the National Academy of Sciences

Volume

110

Issue

19

Start Page

E1706

DOI

10.1073/pnas.1304296110

Abstract

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.

Copyright/Permission Statement

© 2013 National Academy of Sciences.

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Date Posted:19 April 2018

This document has been peer reviewed.