Restoration of cone vision in a mouse model of achromatopsia

Author:  ["John J Alexander","Yumiko Umino","Drew Everhart","Bo Chang","Seok H Min","Qiuhong Li","Adrian M Timmers","Norman L Hawes","Ji-jing Pang","Robert B Barlow","William W Hauswirth"]

Publication:  Nature Medicine

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Tags:     Medicine

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 Gnat2cpfl3 mouse model of achromatopsia.

Cite this article

Alexander, J., Umino, Y., Everhart, D. et al. Restoration of cone vision in a mouse model of achromatopsia. Nat Med 13, 685–687 (2007). https://doi.org/10.1038/nm1596

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