Human Hematopoietic Stem Cell Center of Excellence

这项新的NIDDK P30提案寻求在宾夕法尼亚大学(UPenn)和费城儿童医院(CHOP)的联合校区建立血液学卓越中心。我们相信，良性血液学领域的研究和教育已经达到了UPenn/CHOP的临界值。这项提案的13名调查人员得到了美国国立卫生研究院的充足资金，并已出版，并有多篇合著论文和拨款。我们最近在研究和临床基础设施方面由研究人员推动的增强措施包括：a)建立人类胚胎干细胞中心(2008)；b)建立一个主要的血液中心，专注于加强良性血液病的“床边”研究(2009)；以及c)招募了两名新的备受尊敬的研究人员，以建立一个全面的成人和儿童骨髓衰竭计划，以协调患者护理以及基础和翻译研究。我们的教育工作得到了两大NIH培训赠款的支持：NIDDK良性造血T32奖和NHLBI K12良性血液学奖。随着宾夕法尼亚大学/CHOP血液学研究人员、研究项目、临床项目和受训人员数量的增加，将我们的努力集中到一个具有凝聚力、协同性的整体中变得越来越重要。为了满足这一需求，目前的应用程序寻求支持，以建立和维护一个关于良性人类造血和相关疾病的多研究者协作计划。科学工作将涉及三个相互交织的焦点：1)促进我们对正常造血和BMF综合征的理解，2)利用这些知识开发针对血红蛋白疾病和BMF综合征的新治疗方法，以及3)利用在(1)中获得的知识开发用于治疗血液疾病的基于细胞的新疗法。我们相信，这一幸运的P30 RFA提供了机会来开发和增强我们目前在良性血液学方面的优势。因此，我们寻求开发一个有组织的造血计划，提供集中的最先进的核心设施技术支持，提供吸引年轻研究人员的试点计划，并组织教育活动。我们预计，5年的这种支持将提高我们的生产率，这体现在良性造血领域的重要出版物、更多的协同研究努力、专注于相关领域的教职员工和研究员数量的增长以及我们基于NIH的资金的进一步增加，特别是多名研究人员的资助。

This new NIDDK P30 proposal seeks to establish a Center of Excellence in Hematology 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 a UPENN/CHOP. The 13 Investigators of this proposal are well funded by the NIH and published and have multiple co-authored papers and grants. Recent investigator driven enhancements in our research and clinical infrastructures include establishing: a) a Human Embryonic Stem Cell Center (2008); b) an overarching Blood Center focused on enhancing "bench-to-bedside" research in benign hematology (2009); and c) have recruited two new highly regarded investigators to establish a Comprehensive Adult and Pediatric Bone Marrow Failure Program to coordinate patient care as well as basic and translational research. Our educational efforts 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, the current application seeks support to establish and maintain a multi-investigator, collaborative program on benign human hematopoiesis and associated diseases. Scientific efforts will 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 the hemoglobinopathies and BMF syndromes, as well as 3) using the knowledge gained under (1) to develop novel cellular-based therapeutics for the treatment of hematologic disorders. We believe that this propitious P30 RFA offers the opportunity to exploit and enhance our current strengths in benign hematology. Accordingly, we seek to develop an organized hematopoiesis program that will provide centralized state-of-the-art core facility technical support, offer pilot programs to attract young investigators and organize educational events. We anticipate that 5 years of such 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 a further increase in our NIH-based funding, particularly multi-investigator grants.