Cincinnati Center for Excellence in Molecular Hematology

分子和细胞疗法在可移植造血细胞中的成功应用，包括 造血干细胞有望终生治愈骨髓源性遗传性疾病 细胞。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基因/环境相互作用影响细胞的疾病 来源于造血干细胞。我们认为，要实现这一目标，我们必须了解基本情况 在体外和体内影响淋巴-造血干细胞行为的生物学过程。我们设想 这一长期目标将通过跨学科的方法实现，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中及时合理地使用翻译研究 设置。辛辛那提儿童医院医学中心，辛辛那提大学 医学(UC)，已经开发了一个知识环境，将优秀的病毒专业知识聚集在一起 载体技术，干细胞生物学，免疫细胞生物学，信号转导基础研究和临床护理 血液学、过敏/免疫学和造血干细胞移植。这一焦点在很大程度上 在过去10年中，CCHMC和加州大学在这些领域招聘了50多名教员，加强了这一能力。这个 基础研究领域通过一个新开发的独特的翻译研究倡议联系在一起，该倡议 促进了跨学科的互动，并开发了支持最先进技术的基础设施 细胞和基因治疗方面的试验。因此，在这次申请新的CEMH中，我们寻求巩固令人兴奋的和 CCHMC在血液学和相关天然免疫/免疫学方面的研究急剧增长 这一中心拨款机制的共享服务和管理结构。CCEMH寻求支持 完善的翻译研究核心，补充了一系列杰出的基础科学核心 在CCHMC。这些核心正在发挥作用，以支持创新研究和临床试验，包括在当地和 国内和国际上的一些病例，涉及血液学和相关免疫疾病。相关的 核心包括一个小鼠异种移植/转基因核心，它维护专门的小鼠品系和 提供小鼠移植和转基因服务，这是一个流式细胞术核心，包括最先进的 支持尖端技术的基因组和遗传学核心--血液学流动分析和分选功能 血液病的基因组分析和干/祖细胞的正常遗传特征，以及 翻译核心，涵盖体外操作和收集人类细胞产品等服务， 兰蒂病毒和逆转录病毒的生产，以及复杂的临床前检测开发。最后，这笔核心赠款 寻求对试点/可行性方案的支持，该方案将重点放在非常成功的机构同行评审试点项目上 血液学和相关免疫学研究的项目倡议和主办的增强计划 该中心的研讨会和年度务虚会。

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 ofthe 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 ofthe center.