Cincinnati Center for Excellence in Molecular Hematology

辛辛那提分子血液学卓越中心

基本信息

批准号：
8704524
负责人：
YI ZHENG
金额：
$ 4.73万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2010
资助国家：
美国
起止时间：
2010-09-30 至 2015-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8704524
关键词：
Academic Medical Centers Affect Area Basic Science Biological Process Bone Marrow Diseases Bone Marrow Transplantation Cancer Center Support Grant Caring Cell Adhesion Molecules Cell Therapy Cell Volumes Cells Cellular biology Child Clinical Clinical Immunology Clinical Management Clinical Research Clinical Trials Code Collection Complement Complex Comprehensive Health Care Development Disease Doctor of Philosophy Environment Erythrocytes Erythropoiesis Experimental Hematology FDA approved Faculty Fanconi&apos s Anemia Flow Cytometry Foundations Functional disorder Funding Future Genes Genetic Genomics Goals Grant Growth Growth Factor Hematological Disease Hematology Hematopoiesis Hematopoietic Hematopoietic Stem Cell Transplantation Hematopoietic stem cells Hemoglobinopathies Home environment Human Hypersensitivity Immune Immune system Immunity Immunobiology Immunologic Deficiency Syndromes Immunologics Immunology In Vitro Individual Inherited Innovative Therapy Investments Lentivirus Vector Leukocytes Life Link Medical center Medicine Methods Molecular Molecular Biology Mouse Strains Mus Myelogenous Myeloid Cells National Heart, Lung, and Blood Institute Natural Immunity North America Pancytopenia Participant Pathogenesis Pathway interactions Patients Pediatric Hospitals Peer Review Phase Physicians Physiological Pilot Projects Pneumonia Process Production Publications Rare Diseases Regulation Research Research Infrastructure Research Personnel Research Support Retroviridae Scientist Services Sickle Cell Sickle Cell Anemia Side Signal Pathway Signal Transduction Societies Sorting - Cell Movement Stem cells Structure Subfamily lentivirinae Syndrome Technology Therapeutic Transfusion Transgenic Organisms Translating Translational Research Transplantation United States United States National Institutes of Health Universities Viral Vector Virus Wiskott-Aldrich Syndrome assay development base cell behavior cellular transduction chemokine clinical care college cytokine design gene therapy genetic manipulation immunohematology in vivo inhibitor/antagonist innovation member mouse model oncology patient population pre-clinical pre-clinical research preclinical study programs rho GTP-Binding Proteins small molecule stem stem cell biology trait translational study vector

项目摘要

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.

分子和细胞疗法成功应用于可移植造血细胞（包括造血干细胞），有望终身治愈骨髓来源细胞的遗传性疾病。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基因/环境相互作用影响造血干细胞来源的疾病。我们相信，为了实现这一目标，我们必须了解影响淋巴造血干细胞体外和体内行为的基本生物过程。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的跨学科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来实现。辛辛那提大学医学院 (UC) 的辛辛那提儿童医院医疗中心 (CCHMC) 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、信号传导基础研究和临床护理方面的杰出专业知识。血液学、过敏/免疫学和造血干细胞移植。过去 10 年，CCHMC 和 UC 在这些领域招聘了 50 多名教员，大大加强了这一重点。基础研究领域通过新开发的独特转化研究计划联系在一起，该计划促进了跨学科互动，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，在新 CEMH 的申请中，我们寻求利用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/免疫学研究令人兴奋和显着的增长。 CCEMH 致力于支持完善的转化研究核心，以补充 CCHMC 一系列出色的基础科学核心。这些核心的作用是支持本地以及在某些情况下在国内和国际上在血液学和相关免疫疾病方面的创新研究和临床试验。相关核心包括小鼠异种移植/转基因核心，它维护专门的小鼠品系并提供小鼠移植和转基因服务，流式细胞术核心，包括最先进的血液学流分析和分选功能，基因组学和遗传学核心，支持血液疾病和干细胞/祖细胞正常遗传特征的尖端基因组分析，以及涵盖人类细胞产品的离体操作和收集、慢病毒和逆转录病毒生产等服务的转化核心，和复杂的临床前检测开发。最后，这项核心拨款寻求对试点/可行性计划的支持，该计划重点关注血液学和相关免疫学研究方面非常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加强计划。