Restoration of cone vision in a mouse model of achromatopsia.
Document Type
Article
Publication Date
2007
Keywords
Cones-(Retina), Disease-Models-Animal, Eye-Proteins, Gene-Therapy, Heterotrimeric-GTP-Binding-Proteins, Mice-Transgenic
First Page
685
Last Page
687
JAX Source
Nat Med 2007 Jun; 13(6):685-7.
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.
Recommended Citation
Alexander JJ,
Umino Y,
Everhart D,
Chang B,
Min SH,
Li Q,
Timmers AM,
Hawes NL,
Pang JJ,
Barlow RB,
Hauswirth WW.
Restoration of cone vision in a mouse model of achromatopsia. Nat Med 2007 Jun; 13(6):685-7.