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Nat Med. 2007 Jun;13(6):685-7. Epub 2007 May 21.

Restoration of cone vision in a mouse model of achromatopsia.

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1
Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, Florida 32610, USA.

Abstract

Loss of cone function in the central retina is a pivotal event in the development of severe vision impairment for many prevalent blinding diseases. Complete achromatopsia is a genetic defect resulting in cone vision loss in 1 in 30,000 individuals. Using adeno-associated virus (AAV) gene therapy, we show that it is possible to target cones and rescue both the cone-mediated electroretinogram response and visual acuity in the Gnat2 ( cpfl3 ) mouse model of achromatopsia.

PMID:
17515894
PMCID:
PMC3985124
DOI:
10.1038/nm1596
[Indexed for MEDLINE]
Free PMC Article
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