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.html Application format and review criteria are specified in the RFA. The deadline for submission is Wednesday, April 5, 2017. Applications are restricted to faculty of Penn, CHOP, and The Wistar Institute only. Questions regarding the scientific content of potential projects can be directed to Laura Richman at richmanl@upenn.edu; administrative queries should be sent to Samantha Charleston at scharle@upenn.edu. Thank you, James M. Wilson, MD, PhD Rose H. Weiss Professor and Director, Orphan Disease Center Professor of Medicine and Pediatrics Director, Gene Therapy Program