December 2007
Volume 7, Issue 15
Free
OSA Fall Vision Meeting Abstract  |   December 2007
Gene therapy treatment of color blindness in adult primates
Author Affiliations
  • Katherine Mancuso
    Department of Ophthalmology, Medical College of Wisconsin
  • Jay Neitz
    Department of Ophthalmology, Medical College of Wisconsin
  • William W. Hauswirth
    Department of Ophthalmology and Powell Gene Therapy Center, University of Florida, Gainesville
  • Thomas B. Connor
    Department of Ophthalmology, Medical College of Wisconsin
  • Maureen Neitz
    Department of Ophthalmology, Medical College of Wisconsin
Journal of Vision December 2007, Vol.7, 15. doi:10.1167/7.15.15
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      Katherine Mancuso, Jay Neitz, William W. Hauswirth, Thomas B. Connor, Maureen Neitz; Gene therapy treatment of color blindness in adult primates. Journal of Vision 2007;7(15):15. doi: 10.1167/7.15.15.

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Abstract

We have used gene therapy in which an adeno-associated viral vector containing a human photopigment gene was injected in the eyes of adult dichromatic squirrel monkeys, with the intent of adding red-green color vision. The goal was to exploit the capricious nature of viral infection to transduce only a subset of cones, producing a retinal region with two randomly-interspersed cone types absorbing in the middle-to-long wavelengths. To test color vision, we adapted the Cambridge Colour Test for use with animals. The monkeys' pre-therapy behavior was highly reliable, and they always failed to make “red-green” color discriminations as predicted from their known cone complement. After treatment, the monkeys showed marked improvement in red-green color vision. Conventional wisdom has held that “critical periods” exist for the development of new visual capacities. This has raised the concern that treating adults with congenital conditions could be impossible. To the contrary, here, adult monkeys that had been red-green colorblind from birth obtained a new dimension of color vision as the result of adding a third photopigment. The successful treatment of adult primates with a congenital color vision deficiency is encouraging for the possibility of gene therapy to treat a variety of inherited vision disorders in humans.

Mancuso, K. Neitz, J. Hauswirth, W. W. Connor, T. B. Neitz, M. (2007). Gene therapy treatment of color blindness in adult primates [Abstract]. Journal of Vision, 7(15):15, 15a, http://journalofvision.org/7/15/15/, doi:10.1167/7.15.15. [CrossRef]
Footnotes
 This work was supported by the National Institutes of Health grants R03EY014056, R01EY016861, R01EY11123 (W. W. H.), Research Training Program in Vision Science Grant T32EY014537, NEI Core Grant for Vision Research EY01981, the Harry J. Heeb Foundation, and Research to Prevent Blindness.
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