Please refer to the RFA for instructions and requirements for this submission located on our website at: http://www.med.upenn.edu/orphandisease/rare-disease-overview.shtml Application format and review criteria are specified in the RFA. The deadline for submission of Pre-applications is September, 15 2014 and are restricted to non-profit institutions and foundations. Questions regarding the scientific content of potential projects can be directed to James M. Wilson, M.D., Ph.D. at wilsonjm@mail.med.upenn.edu; administrative queries should be sent to Linda Aversa-Caldwell at laversa@mail.med.upenn.edu. Thank you, James M. Wilson, M.D., Ph.D. Research Director, Center for Orphan Disease and Research and Therapy Director, Gene Therapy Program Professor, Department of Pathology & Laboratory Medicine, Division of Transfusion Medicine