Brief Communication abstract
Nature Medicine 13, 685 - 687 (2007)
Published online: 21 May 2007 | doi:10.1038/nm1596
Restoration of cone vision in a mouse model of achromatopsia
John J Alexander1, Yumiko Umino2, Drew Everhart2, Bo Chang3, Seok H Min4, Qiuhong Li4, Adrian M Timmers4,5, Norman L Hawes3, Ji-jing Pang4, Robert B Barlow2 & William W Hauswirth1,4
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.
- Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, Florida 32610, USA.
- Center for Vision Research, Department of Ophthalmology, State University of New York Upstate Medical University, Syracuse, New York 13210, USA.
- The Jackson Laboratory, Bar Harbor, Maine 04609, USA.
- Department of Ophthalmology, University of Florida College of Medicine, Gainesville, Florida 32610, USA.
- Present address: Alcon Research Ltd., Fort Worth, Texas 76134, USA.
Correspondence to: William W Hauswirth1,4 e-mail: hauswrth@eye.ufl.edu
MORE ARTICLES LIKE THIS
These links to content published by NPG are automatically generated.
NEWS AND VIEWS
Unraveling the ribbon synapseNature Neuroscience News and Views (01 Aug 2008)
The sights along route 65Nature Genetics News and Views (01 Sep 2001)
RESEARCH
Restoration of cone vision in a mouse model of achromatopsiaNature Medicine Brief Communication
Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood BlindnessMolecular Therapy Original Article
See all 73 matches for Research
