Restoration of cone vision in a mouse model of achromatopsia.
basic_science · Level V
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- Record sourced from PubMed, PMID 17515894.
- Also identified by PMC identifier 3985124.
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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.
Medical subject headings
- Color Vision Defects
- Disease Models, Animal
- Genetic Therapy
- Retinal Cone Photoreceptor Cells