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Gene Therapy Improves Vision in Patients with Congenital Retinal Disease
Date:4/27/2008

a generous gift from the F.M. Kirby Foundation, which has provided continuous support for the ongoing research for the past 14 years. The F.M. Kirby Center was the first molecular biology center devoted to developing gene therapy for hereditary causes of vision loss. The Center serves as home to the laboratories of seven investigators who conduct research on the cellular and molecular biology of eye disease and visual function. Current studies in the F.M. Kirby Center include evaluations of the molecular genetics and pathogenetic mechanisms involved in optic nerve disease and inherited retinal and macular degenerations, cell biology studies of photoreceptor sensory cilia, delineation of mechanisms underlying the light responses of rods and cones, gene discovery of complex and monogenic disorders, development of methods with which to non-invasively monitor retinal and visual function in animal models and humans, and design of novel methods with which to image retinal cells. (http://www.uphs.upenn.edu/news)

The Department of Ophthalmology (Scheie Eye Institute) at Penn is a world leader in patient care and eye and vision research. In 2006, the most recent year for which published data are available, Scheie Eye Institute was the #1 recipient of eye research funds from the National Eye Institute, National Institutes of Health among all departments of ophthalmology in the United States. Currently the National Eye Institute is funding a 46-site randomized clinical trial to evaluate treatment strategies for age-related macular degeneration coordinated by faculty at Scheie Eye Institute at Penn.

About The Telethon Institute of Genetics and Medicine (TIGEM): TIGEM is a local and international reference for research on human genetic diseases. It was created in 1994 by the Telethon Foundation, one of Italy's major non-profit organizations, to promote the advancement of research aimed at the diagnosis
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SOURCE The Children's Hospital of Philadelphia; University ofPennsylvania
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