1. Restoration of cone vision in a mouse model of achromatopsia.
- Author
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Alexander, John J., Umino, Yumiko, Everhart, Drew, Bo Chang, Min, Seok H., Qiuhong Li, Timmers, Adrian M., Hawes, Norman L., Ji-jing Pang, Barlow, Robert B., and Hauswirth, William W.
- Subjects
ACHROMATISM ,VISION disorders ,RETINA ,GENETIC disorders ,ADENOVIRUSES ,DNA viruses ,GENE therapy - 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. [ABSTRACT FROM AUTHOR]- Published
- 2007
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