Cincinnati Center for Excellence in Molecular Hematology

分子和细胞疗法的成功应用于可移植的造血细胞，包括造血干细胞，有望终生治愈骨髓源性细胞的遗传性疾病。辛辛那提分子血液学卓越中心(CCEMH)的长期目标是在分子水平上了解和纠正影响造血干细胞来源的细胞的基因/环境相互作用的疾病。我们认为，为了实现这一目标，我们必须了解在体外和体内影响淋巴-造血干细胞行为的基本生物学过程。我们预计这一长期目标将通过使用最先进的分子和细胞生物学方法以及在临床前和临床环境中及时和合理地使用转化性研究的跨学科方法来实现。辛辛那提儿童医院医疗中心(CCHMC)、辛辛那提大学医学院(UC)建立了一个智力环境，汇集了病毒载体技术、干细胞生物学、免疫细胞生物学、信号基础研究和血液学、过敏/免疫学和造血干细胞移植方面的临床护理方面的杰出专业知识。在过去的10年里，CCHMC和UC在这些领域招聘了50多名教师，大大加强了这一重点。基础研究领域通过新制定的独特的转化研究倡议联系在一起，该倡议促进了跨学科互动，并建立了支持细胞和基因疗法最先进试验的基础设施。因此，在这次申请新的CEMH中，我们寻求巩固CCHMC在血液学和相关天然免疫/免疫学方面令人兴奋和戏剧性的研究增长，利用该中心赠款机制的共享服务和管理结构。CCEMH寻求支持一个完善的翻译研究核心，作为CCHMC一系列杰出的基础科学核心的补充。这些中心正在发挥作用，支持当地的创新研究和临床试验，在某些情况下，在国家和国际上支持血液学和相关的免疫疾病方面的研究和临床试验。相关的核心包括维护专门的小鼠品系并提供小鼠移植和转基因服务的小鼠异种移植/转基因核心；包括最先进的血液学流动分析和分选能力的流式细胞术核心；支持血液疾病的尖端基因组分析和干细胞/祖细胞的正常遗传特征的基因组学和遗传学核心；以及涵盖服务的翻译核心，包括体外操作和收集人类细胞产品、Lenti和逆转录病毒生产以及复杂的临床前分析开发。最后，这笔核心赠款寻求支持一项试点/可行性计划，该计划将一个非常成功的机构同行评审的试点项目倡议集中到血液学和相关的免疫学研究中，并寻求支持该中心举办研讨会和年度务虚会的增强计划。

The successful application of molecular and cell therapeutics to transplantable hematopoietic cells, including hematopoietic stem cells, holds the promise of life-long cures for inherited diseases of bone marrow-derived cells. The long term goal of the Cincinnati Center of Excellence in Molecular Hematology (CCEMH) is to understand and correct at the molecular level diseases with gene/environmental interactions affecting cells derived from hematopoietic stem cells. We believe that to attain this goal, we must understand basic biological processes that affect lympho-hematopoietic stem cell behavior in vitro and in vivo. We envision this long term goal will be accomplished by inter-disciplinary approaches using state-of-the-art molecular and cell biology methods along with timely and rational use of translational studies in the preclinical and clinical settings. Cincinnati Children's Hospital Medical Center (CCHMC), University of Cincinnati College of Medicine (UC), has developed an intellectual environment that brings together outstanding expertise in virus vector technology, stem cell biology, immune cell biology, basic research in signaling and clinical care in hematology, allergy/immunology and hematopoietic stem cell transplantation. This focus has been greatly strengthened by recruitment of over 50 faculty in these areas in the last 10 years at CCHMC and UC. The areas of basic research are linked by a newly developed and unique Translational Research Initiative, which has facilitated inter-disciplinary interactions and developed an infrastructure that is supporting state-of-the-art trials in cell and gene therapy. Thus, in this application for a new CEMH, we seek to solidify exciting and dramatic growth of research in hematology and related innate immunity/immunology at CCHMC using the shared services and administrative structure of this center grant mechanism. The CCEMH seeks to support a well developed translational research core which complements an outstanding array of basic science cores at CCHMC. These cores are functioning to support innovative research and clinical trials both locally and, in some cases nationally and internationally, in hematology and related immune-based diseases. The relevant cores include a Mouse Xenotransplant/transgenic Core, which maintains specialized mouse strains and provides mouse transplant and transgenic services, a Flow Cytometry Core that includes state-of-the-art hematology flow analysis and sorting capabilities, a Genomics & Genetics Core that support cutting edge genomic analysis of hematologic diseases and normal genetic traits of stem/progenitor cells, and a Translational Core that covers services such as ex vivo manipulation and collection of human cell products, lenti- and retrovirus production, and sophisticated preclinical assay development. Finally, this core grant seeks support for a Pilot/Feasibility program that focuses a highly successful institutional peer-reviewed pilot project initiative into hematologic and related immunologic research and an enhancement program in hosting seminars and annual retreat of the center.