Neuroscience

Investigators at University of Oklahoma Describe Research in Neuroscience

Published in Gene Therapy Weekly, January 26th, 2012

2012 JAN 26 -- "RPE65 is an abundantly expressed protein within the retinal pigment epithelium (RPE) of the eye that is required for retinoid metabolism to support vision. Its genetic mutations are linked to the congenital disease Leber congenital amaurosis Type 2 (LCA2) characterized by the early onset of central vision loss," investigators in Oklahoma City, Oklahoma report.

"Current gene therapy trials have targeted restoration of functional RPE65 within the RPE of these patients with some success. Recent data show that RPE65 is also present within mouse cones to promote function. In this study, we evaluated the presence of RPE65 in human cones and...

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Source: Gene Therapy Weekly (2012-01-26)

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