Peer-reviewed veterinary case report
Restoration of cone vision in a mouse model of achromatopsia.
- Journal:
- Nature medicine
- Year:
- 2007
- Authors:
- Alexander, John J et al.
- Affiliation:
- Department of Molecular Genetics and Microbiology · United States
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.
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Search related cases →Original publication: https://pubmed.ncbi.nlm.nih.gov/17515894/