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Article

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2015

Banin Eyal, Gootwine Elisha, Obolensky Alexey, Ezra-Elia Raaya, Ejzenberg Ayala, Zelinger Lina, Honig Hen, Rosov Alexander, Yamin Esther, Sharon Dror, Averbukh Edward, Hauswirth William W, Ofri Ron

Abstract excerpt

Achromatopsia is a hereditary form of day blindness caused by cone photoreceptor dysfunction. Affected patients suffer from congenital color blindness, photosensitivity, and low visual acuity. Mutations in the CNGA3 gene are a major cause of achromatopsia, and a sheep model of this disease was recently characterized by our group. Here, we report that unilateral subretinal delivery of an adeno-associated virus...

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Source

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Identifiers

PMID: 26087757
DOI: 10.1038/mt.2015.114
PMCID: PMC4817879

Genes

CNGA3
ERG

Provenance

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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Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia.PMID 26087757Read ↗

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