Restoration of cone vision in a mouse model of achromatopsia |
| |
| Authors: | Alexander John J Umino Yumiko Everhart Drew Chang Bo Min Seok H Li Qiuhong Timmers Adrian M Hawes Norman L Pang Ji-Jing Barlow Robert B Hauswirth William W |
| |
| Institution: | Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, Florida 32610, USA. |
| |
| 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. |
| |
| Keywords: | |
| 本文献已被 PubMed 等数据库收录！ |
|
