Restoration of cone vision in a mouse model of achromatopsia.
Cones-(Retina), Disease-Models-Animal, Eye-Proteins, Gene-Therapy, Heterotrimeric-GTP-Binding-Proteins, Mice-Transgenic
Nat Med 2007 Jun; 13(6):685-7.
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.
Alexander, J J.; Umino, Y; Everhart, D; Chang, B; Min, S H.; Li, Q; Timmers, A M.; Hawes, N L.; Pang, J J.; Barlow, R B.; and Hauswirth, W W., "Restoration of cone vision in a mouse model of achromatopsia." (2007). Faculty Research 2000 - 2009. 1628.