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的临界质量。该提案的13名研究人员得到了美国国立卫生研究院的大量资助，并发表了多篇共同撰写的论文和赠款。最近研究者推动了我们的研究和临床基础设施的改进，包括建立：a)人类胚胎干细胞中心（2008年）；b)一个全面的血液中心，专注于加强良性血液学“从实验室到床边”的研究（2009年）；c)招募了两名新的备受推崇的研究者来建立一个综合成人和儿童骨髓衰竭项目，以协调患者护理以及基础和转化研究。我们的教育工作得到了两项主要NIH培训补助金的支持：NIDDK良性造血T32和NHLBI良性血液学K12奖。随着UPENN/CHOP血液学研究人员、研究项目、临床项目和培训生数量的增加，将我们的努力集中到一个有凝聚力的、协同的整体中变得越来越重要。为了满足这一需求，目前的申请寻求支持，以建立和维持一个多研究者合作的良性人类造血和相关疾病项目。科学工作将涉及三个相互交织的焦点：1)推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病和BMF综合征的新治疗方法，以及3)利用第(1)项下获得的知识开发用于治疗血液病的新型基于细胞的治疗方法。我们相信，这一有利的P30 RFA为开发和增强我们目前在良性血液学方面的优势提供了机会。因此，我们寻求开发一个有组织的造血项目，该项目将提供集中的最先进的核心设施技术支持，提供试点项目以吸引年轻的研究人员并组织教育活动。我们预计，这5年的支持将提高我们的生产力，证据是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师和研究员数量的增长，以及我们nih资助的进一步增加，特别是多研究者资助。

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.