Regulating hematopoietic stem cells and hematopoiesis for clinical efficacy

该计划的总体目标是在分子水平上理解和纠正影响多能造血干细胞衍生细胞的遗传疾病。我们相信，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转化的努力。该申请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）、新生儿学、血管和发育生物学方面具有多样化但互补的经验。这个小组与许多共同撰写的出版物以及跨越基础、转化和临床研究的资助项目高度协作和互动。在这个CEMH提交中提出的核心已经发展并适应了这个项目的当前需求。这些包括：流式细胞术、动物、成像和血管生成/内皮/原血管生成核心。所有核心将支持基础研究和转化研究，这些研究是CEMH使命的基础，并将促进新发现进入人体试验。该计划还包括一个试点和可行性项目，部分由大学基金资助，以加强对年轻研究人员的培训，提高他们成功竞争校外（如NIH）资助的能力。总之，CEMH代表了一个重要的关键核心组合，需要继续和加强正在进行的基础工作，并将其落实到位，我们认为未来五年将需要这些支持。

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.