1. Restoration of cone vision in a mouse model of achromatopsia.
- Author
-
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
- Full Text
- View/download PDF