On Friday, April 6, 2007, a symposium was held at the Coleman Center in New York under the auspices of the Foundation Fighting Blindness. The symposium was co-chaired by Gerald A. Fishman, M.D., and Rando Allikmets, Ph.D. A list of participants and agenda is included at the end of the document.

The intended purpose of the meeting was to address issues relevant to moving pre-clinical studies of Stargardt disease into clinical trials.

This brochure provides you with some general background on gene transfer, what it is and how it works. It also includes two lists of questions. The first list suggests questions that you might want to ask your health provider or a member of the research team. The second list can serve as a guide to help you decide whether this study is right for you.

The National Neurovision Research Institute (NNRI) hosted the Symposium to discuss, evaluate, and promote translational research for the development of preventions, treatments, and cures for retinal degenerative diseases. NNRI was established in 2002 to expedite the translation of laboratory-based research into clinical trials for treatment of hereditary orphan retinal diseases. The Symposium was a key strategic step by the Institute to develop bridges of communication between scientific, clinical, governmental, pharmaceutical and financial communities, and to encourage clinical trials of new candidate drugs and drug delivery systems for orphan retinal diseases.

Stem cells have the potential to replace damaged and/or dead photoreceptors and retinal pigment epithelial cells. Advances in this technology will facilitate the preservation and restoration of useful vision in individuals affected by inherited orphan retinal degenerative diseases and geographic atrophy.