Cincinnati Center for Excellence in Molecular Hematology

分子和细胞疗法在可移植造血细胞（包括造血干细胞）上的成功应用，为骨髓源性细胞遗传性疾病的终身治疗带来了希望。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理解和纠正影响来自造血干细胞的细胞的基因/环境相互作用疾病。我们认为，为了实现这一目标，我们必须了解影响体内和体外淋巴造血干细胞行为的基本生物学过程。我们设想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合理地使用转化研究的跨学科方法来实现。辛辛那提儿童医院医学中心（CCHMC），辛辛那提大学医学院（UC），开发了一个智力环境，汇集了病毒载体技术，干细胞生物学，免疫细胞生物学，血液学，过敏/免疫学和造血干细胞移植的信号传导和临床护理基础研究方面的杰出专业知识。在过去的10年里，CCHMC和UC在这些领域招募了50多名教师，大大加强了这一重点。基础研究领域由新开发的独特的转化研究计划联系起来，该计划促进了跨学科的互动，并开发了支持最先进的细胞和基因治疗试验的基础设施。因此，在这个新的CEMH申请中，我们寻求通过该中心资助机制的共享服务和管理结构，巩固CCHMC血液学和相关先天免疫/免疫学研究的令人兴奋和戏剧性的增长。CCEMH旨在支持一个发达的转化研究核心，以补充CCHMC优秀的基础科学核心。这些核心正在发挥作用，支持在血液学和相关免疫疾病方面的创新研究和临床试验，在某些情况下，还支持在国内和国际上进行的创新研究和临床试验。相关核心包括一个小鼠异种移植/转基因核心，该核心负责维护专门的小鼠品系并提供小鼠移植和转基因服务；一个流式细胞术核心，包括最先进的血液学流分析和分选能力；一个基因组学和遗传学核心，支持对血液学疾病和干细胞/祖细胞的正常遗传性状进行尖端基因组分析。以及翻译核心，涵盖诸如离体操作和人类细胞产品收集、慢体和逆转录病毒生产以及复杂的临床前分析开发等服务。最后，这项核心拨款寻求对试点/可行性计划的支持，该计划将重点放在血液学和相关免疫学研究中高度成功的机构同行评审试点项目倡议，以及举办研讨会和中心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.