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Article

Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness.

PLoS medicine - 1 Jun 2006

Williams Melissa L, Coleman Jason E, Haire Shannon E, Aleman Tomas S, Cideciyan Artur V, Sokal Izabel, Palczewski Krzysztof, Jacobson Samuel G, Semple-Rowland Susan L

Abstract excerpt

BACKGROUND: Leber congenital amaurosis (LCA) is a genetically heterogeneous group of retinal diseases that cause congenital blindness in infants and children. Mutations in the GUCY2D gene that encodes retinal guanylate cyclase-1 (retGC1) were the first to be linked to this disease group (LCA type 1 [LCA1]) and account for 10%-20% of LCA cases. These mutations disrupt synthesis of cGMP in photoreceptor cells, a...

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Identifiers

PMID: 16700630
DOI: 10.1371/journal.pmed.0030201
PMCID: PMC1463903

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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