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 of LOIs is September, 18, 2017 at 8:00pm EST. Applications are restricted to non-profit institutions and foundations. Questions regarding the scientific content of potential projects can be directed to Monique Molloy at moniquek@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