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Genetically engineered large animal model for studying cone photoreceptor survival and degeneration in retinitis pigmentosa

Nature Biotechnology volume 15, pages 965–970 (1997)Cite this article

Abstract

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.

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Author information

Authors and Affiliations

  1. Department of Animal Science, North Carolina State University, Raleigh, NC

    Robert M. Petters, Curtis A. Alexander, Kevin D. Wells, E. Bruce Collins, Jeffrey R. Sommer, Maria R. Blanton, Guadalupe Rojas & William L. Flowers

  2. Department of Ophthalmology, Duke University School of Medicine, Durham, NC

    Ying Hao & Fulton Wong

  3. Departments of Neurobiology and Pathology, Duke University School of Medicine, Durham, NC

    Fulton Wong

  4. Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA

    Eyal Banin, Artur V. Cideciyan & Samuel G. Jacobson

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  1. Robert M. Petters
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  2. Curtis A. Alexander
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Correspondence to Fulton Wong.

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Petters, R., Alexander, C., Wells, K. et al. Genetically engineered large animal model for studying cone photoreceptor survival and degeneration in retinitis pigmentosa. Nat Biotechnol 15, 965–970 (1997). https://doi.org/10.1038/nbt1097-965

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