Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.

Recessive mutations in guanylate cyclase-1 (Gucy2d) are associated with severe, early onset Leber congenital amaurosis-1(LCA1). Gucy2d encodes guanylate cyclase (GC1) is expressed in photoreceptor outer segment membranes and produces cGMP in these cells. LCA1 patients present in infancy with severel...

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Bibliographic Details
Main Authors: Shannon E Boye, Sanford L Boye, Jijing Pang, Renee Ryals, Drew Everhart, Yumiko Umino, Andy W Neeley, Joseph Besharse, Robert Barlow, William W Hauswirth
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2010-06-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC2892468?pdf=render