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Translational Research

Translational research transforms scientific discoveries arising from laboratory, clinical or population studies into clinically relevant applications to improve health by reducing disease incidence, morbidity and mortality. Owing to a dramatically increased understanding of disease mechanisms, NEI's translational research program has made significant progress

Gene Transfer: A pioneering, NEI-funded phase I clinical trial published in 2008 found that gene transfer for Leber congenital amaurosis (LCA) resulted in modest visual improvement. This ongoing clinical trial is the result of years of translational research.

Neurotrophic Agents: During the early 1990s, NEI investigators began evaluating the use of neurotrophic agents in animal models of retinitis pigmentosa (RP), a family of retinal degenerative diseases. These naturally occurring proteins had been previously evaluated in a variety of neurodegenerative diseases. These early laboratory studies established that ciliary neurotrophic factor (CNTF) resulted in photoreceptor cell protection in a variety of animal models of RP. In 2011, Neurotech Pharmaceuticals reported positive results from a phase II clinical trial with the use of ciliary neurotrophic factor (CNTF) in age-related macular degeneration. These encouraging results were made possible with robust support from NEI.

Cell Therapy\Regenerative Medicine: Another promising therapy for eye disease is cell transplantation. Some of these approaches have already led to standard, state-of-the-art treatments, such as corneal transplantation. Other investigational treatments are being developed through NEI's translational research efforts.

Last Reviewed: December 2011



Department of Health and Human Services NIH, the National Institutes of Health USA.gov