Cincinnati Cooperative Center of Excellence in Hematology

摘要/摘要： 辛辛那提血液学卓越合作中心(CCCEH)的长期目标是 在分子水平上理解和纠正不同血统的血液疾病。为了实现这一目标， 我们必须了解影响造血干细胞行为的基本生物学过程及其 体外和体内各种分化的后代。我们设想这一长期目标将得到最好的实现。 通过使用最新方法的跨学科和协作方法，以及及时和合理的 对临床前和临床环境的转化性研究的使用。辛辛那提儿童医院医疗 中心(CCHMC)发展了一个丰富而鼓舞人心的智力环境，将 在干细胞生物学、病毒载体技术、免疫血液学细胞方面的杰出专业知识和基础研究 生物学、基因组学和遗传学以及造血；此外，CCCEH在以下领域拥有世界级的专业知识 血液学、免疫学和造血干细胞移植的翻译和临床研究。 在这些领域招聘了30多名教职员工，大大加强了这一重点 CCHMC过去15年的血液学研究，并纳入了超过15个外部血液学 美国各地的研究人员。基础研究与辛辛那提国立卫生研究院资助的临床和翻译科学有关 奖(CTSA)，它促进了跨学科互动，并开发了支持 细胞和基因治疗方面最先进的临床试验。在这里，我们寻求巩固激动人心的系统性增长 在CCHMC的血液学研究，以支持一个国家卓越中心，使用共享的服务和 中央拨款机制的管理结构。CCCEH管理核心支持 一系列卓越的研究核心，共同支持创新的基础和翻译 在非恶性血液学方面的地方和国家研究。我们建议的三个研究核心包括 (1)异种移植和基因组编辑核心，维护专门的小鼠品系，并为小鼠提供 移植和转基因服务；(2)单细胞鉴定和采购核心，包括国家- 最先进的成像以及单细胞捕获和基因表达能力；以及(3)基因交付核心 这包括通过慢病毒和逆转录病毒转导体外操纵血细胞和 ShRNA/sgRNA筛选。最后，该中心寻求对创新丰富计划的支持，该计划旨在促进 中心的协作和教育环境，以及广泛的血液学研究的外展 美国各地的社区。

SUMMARY/Abstract: The long-term goal of the Cincinnati Cooperative Center of Excellence in Hematology (CCCEH) is to understand and correct, at the molecular level, hematological diseases of various lineages. To attain this goal, we must understand basic biological processes that affect behaviors of hematopoietic stem cells and their various differentiated progenies in vitro and in vivo. We envision this long-term goal will be best accomplished by interdisciplinary and collaborative approaches using state-of-the-art methods, along with timely and rational use of translational studies into the preclinical and clinical settings. Cincinnati Children's Hospital Medical Center (CCHMC) has developed a rich and stimulating intellectual environment that brings together outstanding expertise and basic research in stem cell biology, virus vector technology, immunohematologic cell biology, genomics and genetics, and hematopoiesis; in addition, the CCCEH has world-class expertise in translational and clinical research in hematology, immunology and hematopoietic stem cell transplantation. This focus has been greatly strengthened by the recruitment of over 30 faculty members in these areas of hematology research over the last 15 years at CCHMC, and the inclusion of over 15 external hematology researchers around US. Basic research is linked with Cincinnati NIH-funded Clinical and Translational Science Award (CTSA), which has facilitated interdisciplinary interactions and developed an infrastructure that supports state-of-the-art clinical trials in cell and gene therapy. Here we seek to solidify the exciting and systemic growth of research in hematology at CCHMC to support a national center of excellence, using the shared services and administrative structure of the center grant mechanism. The CCCEH Administrative Core supports an exceptional array of research cores, which function together to support innovative basic and translational research both locally and nationally, in non-malignant hematology. Our three proposed research cores include (1) Xenotransplant and Genome Editing Core, which maintains specialized mouse strains and provides mouse transplant and transgenic services; (2) Single Cell Characterization and Procurement Core that includes state- of-the-art imaging as well as single cell capture and gene expression capabilities; and (3) Gene Delivery Core that covers services such as ex vivo manipulation of blood cells by lentivirus and retrovirus transduction and shRNA/sgRNA screening. Finally, this center seeks support for an innovative Enrichment Program that fosters a collaborative and educational environment in the center and for outreach to the broad hematology research community around US.