Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse

BACKGROUND: 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...

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Main Authors: Boye, Shannon E., Boye, Sanford L., Pang, Jijing, Ryals, Renee, Everhart, Drew, Umino, Yumiko, Neeley, Andy W., Besharse, Joseph, Barlow, Robert, Hauswirth, William W.
Format: Artigo
Jezik:Inglês
Izdano: Public Library of Science 2010
Teme:
Research Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2892468/
https://ncbi.nlm.nih.gov/pubmed/20593011
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0011306
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