Regulating hematopoietic stem cells and hematopoiesis for clinical efficacy

该计划的总体目标是在分子水平上了解和纠正影响多能造血干细胞来源的细胞的遗传病。我们认为，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，无论是在体外还是在体内，在基础科学实验室到临床试验的转化努力。本申请中拟议的分子血液学卓越中心(CEMH)成员来自一群资金充足的研究人员，他们在造血和干细胞生物学、病毒介导的基因转移、分子遗传学、病毒学、造血干细胞移植(脐带血、骨髓和细胞因子/试剂介导的动员)、新生儿科以及血管和发育生物学方面拥有不同但互补的经验。这个小组与许多共同撰写的出版物以及跨越基础、翻译和临床研究的资助项目高度协作和互动。CEMH提交的这份意见书中建议的核心已经演变和调整，以适应该计划的当前需求。这些核心包括：流式细胞术、动物、成像和血管生成/内皮/前血管生成核心。所有核心将支持作为CEMH使命基础的基础研究和翻译研究，并将促进将新发现发展为人体试验。该计划还包括一个试点和可行性项目，部分资金来自大学基金，以加强对年轻调查人员的培训，以提高他们成功竞争外部资金(如NIH)的能力。总而言之，妇女地位委员会是继续和加强正在进行的基本工作和落实我们认为今后五年将需要的支助所需的关键核心的重要集合。

The overall goal of this program is to understand and correct, at the molecular level, genetic diseases affecting cells derived from pluripotent hematopoietic stem cells. We believe that to attain this goal, we must understand basic biological processes that affect hematopoietic stem cell behavior both in vitro and in vivo in a basic science laboratory to clinical trial translational effort. The proposed Center of Excellence in Molecular Hematology (CEMH) membership in this application draws from a group of well funded investigators with a diverse but complementary experience in hematopoiesis and stem cell biology, viral mediated gene transfer, molecular genetics, virology, hematopoietic stem cell transplantation (cord blood, bone marrow, and cytokine/reagent mediated mobilization), neonatology and vascular and developmental biology. This group is highly collaborative and interactive with numerous co-authored publications, as well as funded projects that span basic, translational, and clinical research. The Cores proposed in this CEMH submission have evolved and been adapted to the current needs of this program. These include: Flow Cytometry, Animal, Imaging, and Angeogenesis/Endothelial/Proangiogenic Cores. All Cores will support the basic and translational studies that underlying the mission of the CEMH and will facilitate development of new discoveries into human trials. The program also includes a Pilot and Feasibility Project, to be funded in part through University funds in order to enhance the training of young investigators to enhance their ability to successfully compete for extramural (e.g. NIH) funding. Together, the CEMH represents an important assembly of critical cores needed to continue and enhance the basic work in progress and to put in place, support which we believe will be needed over the next five years.