LOIS is due Thursday, March 8th by 5pm EST. Please refer to the RFA for instructions and requirements for this submission located on our website at: http://orphandiseasecenter.med.upenn.edu/grant-opportunities Eligibility All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are eligible to respond to this RFA. Biopharmaceutical companies are not eligible to apply, however, we will consider applications from contract research organizations who provide services that are responsive to the RFA. Questions regarding the scientific content of potential projects can be directed to Dan Lavery at dlavery@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