This NIDDK P30 Center supports a Human Stem Cell Center of Excellence at the joint campuses of the University of Pennsylvania (UPENN) and the Children's Hospital of Philadelphia (CHOP). We believe that research and education in the field of benign hematology has achieved a critical mass at UPENN/CHOP. The investigators supported by this proposal are well-funded by the NIH, and have authored or co-authored multiple papers and grants. Recent investigator-driven enhancements in our research and clinical infrastructures include establishing a Human Embryonic Stem Cell Center; founding an overarching Blood Center, focused on enhancing "bench-to-bedside" research in benign hematology; and recruiting two new highly-regarded investigators to establish a Comprehensive Adult and Pediatric Bone Marrow Failure (BMF) Program to coordinate patient care as well as basic and translational research. Our educational efforts, meanwhile, are supported by two major NIH training grants: an NIDDK benign hematopoiesis T32, and a NHLBI K12 award on benign hematology.

As the number of UPENN/CHOP hematology investigators, research projects, clinical programs, and trainees expand, it becomes increasingly important to focus our efforts into a cohesive, synergistic whole. To address this need, this P30 supported Human Stem Cell Center of Excellence was established and is maintained as a multi-investigator, collaborative program on benign human hematopoiesis and associated diseases. Scientific efforts involve three intertwined foci: 1) advancing our understanding of normal hematopoiesis and of BMF syndromes, 2) using such knowledge to develop novel therapeutic approaches for disorders such as the hemoglobinopathies and BMF syndromes, and 3) using the knowledge gained to develop novel cellular-based therapeutics for the treatment of hematologic disorders.

We believe that this P30 offers investigators the opportunity to exploit and enhance our current strengths in benign hematology by providing discounts on key cores, by supporting Pilot and Feasibility Studies by promising young investigators, by attracting students to the field of hematopoiesis with short-term support, and by supporting a seminar series and annual retreat focused on benign hematopoiesis. We anticipate that this support will enhance our productivity, as evidenced by important publications in the area of benign hematopoiesis, more synergistic research efforts, growth in the number of faculty and fellows focused on relevant areas, and an increase in our NIH-based funding, particularly multi-investigator grants.