Genetically engineered large animal model for studying cone photoreceptor survival and degeneration in retinitis pigmentosa.
Journal Article (Journal Article)
Patients with retinitis pigmentosa (RP) typically develop night blindness early in life due to loss of rod photoreceptors. The remaining cone photoreceptors are the mainstay of their vision; however, over years or decades, these cones slowly degenerate, leading to blindness. We created transgenic pigs that express a mutated rhodopsin gene (Pro347Leu). Like RP patients with the same mutation, these pigs have early and severe rod loss; initially their cones are relatively spared, but these surviving cones slowly degenerate. By age 20 months, there is only a single layer of morphologically abnormal cones and the cone electroretinogram is markedly reduced. Given the strong similarities in phenotype to that of RP patients, these transgenic pigs will provide a large animal model for study of the protracted phase of cone degeneration found in RP and for preclinical treatment trials.
Full Text
Duke Authors
Cited Authors
- Petters, RM; Alexander, CA; Wells, KD; Collins, EB; Sommer, JR; Blanton, MR; Rojas, G; Hao, Y; Flowers, WL; Banin, E; Cideciyan, AV; Jacobson, SG; Wong, F
Published Date
- October 1997
Published In
Volume / Issue
- 15 / 10
Start / End Page
- 965 - 970
PubMed ID
- 9335046
International Standard Serial Number (ISSN)
- 1087-0156
Digital Object Identifier (DOI)
- 10.1038/nbt1097-965
Language
- eng
Conference Location
- United States