Tomas Aleman, M.D.

Scheie Eye Institute, University of Pennsylvania

Project: Retinitis Pigmentosa: Paving the Path to Treatments Through Detailed Understanding of Disease Expression
Summary: Success of ongoing gene therapy trials for Leber Congenital Amaurosis has raised expectations that other forms of inherited retinal degeneration may also be treatable. Detailed characterizations of patients, part of the groundwork that was necessary to initiate these trials, will be performed in other inherited retinal degenerative disorders to increase understanding of human disease expression and mechanism. The knowledge gained will be used to ask specific questions relevant for the planning of future treatment trials for these conditions.