CORE--PRIMATE STEM CELL TRANSPLANTATION

灵长类干细胞移植核心。灵长类干细胞移植核心将为UW/FHCRC PEGT的研究人员和NHLBI的研究人员提供三项主要功能。1)核心将提供非人类灵长类--狒狒(Papio cynocephalus sp.)的造血祖细胞和干细胞群体。和猪尾猕猴(Macaca Nemstrina)，进行体外基因转移实验。2)它将进行体内移植实验，使用从动员后的血液和/或预置的骨髓中提纯的基因修饰的骨髓再生细胞。3)它将进行研究，以测试用于干细胞基因治疗的新的TEM/祖细胞动员计划。当研究人员试图将研究结果从小鼠和其他小动物模型推断到人类的临床环境时，能否获得用于造血干细胞基因治疗的大型动物移植模型可能是一个限制因素。灵长类干细胞移植核心将解决这些限制。该核心将向所有由PET支持的研究人员以及其他NHLBI研究人员开放。在与细胞采购和处理核心的合作下，将向研究人员提供狒狒和猕猴物种的特定血液和骨髓种群，用于体外研究。重要的是，Core将利用威斯康星大学区域灵长类研究中心和FHCRC现有的干细胞移植和基因转移方面的活动。该中心不仅将提供移植动物的护理和管理，还将提供体外造血研究、进行移植、分析动物移植后的细胞和方案开发方面的专业知识。为了最大限度地利用现有动物，将使用竞争性再繁殖策略，以便对不同的方案和媒介进行直接的动物内比较。这一核心为合作研究人员提供相关的大型动物模型的能力应该会极大地促进干细胞生物学的进步，并最终将造血干细胞基因疗法应用于临床。

Primate stem transplantation core. The Primate Stem Cell Transplantation Core will provide three major functions for the investigators of the UW/FHCRC PEGT and for NHLBI investigators. 1) The Core will provide hematopoietic progenitor and stem cell populations from non- human primates, baboons (Papio cynocephalus sp.) and pig-tailed macaques (Macaca nemistrina), for in vitro gene transfer experiments. 2) It will undertake the conduct of in vivo transplantation experiments with gene modified marrow-repopulating cells purified from mobilized blood and/or primed marrow. 3) It will undertake the conduct of studies to test noel tem/progenitor cell mobilization schemes to be used for stem cell gene therapy. Access to large animal transplant models for hematopoietic stem cell gene therapy can be a limiting factor when investigators try to extrapolate findings form the mouse and other small animal models to the human clinical setting. The Primate Stem Cell Transplant Core will function to address these limitations. The Core will be available to all PET supported investigators as well as other NHLBI researchers. In collaboration with the Cell Procurement and Processing Core, specific blood and marrow populations from baboons and macaque species will be provided to investigators for in vitro studies. Importantly, the Core will take advantage of activities in stem cell transplantation and gene transfer in baboons that already exist at the UW-Regional Primate Research Center and FHCRC. The Core not only will provide the care and management of transplanted animals but also will provide expertise on in vitro studies of hematopoiesis, performing transductions, analyzing cells from animals post-transplant and protocol development. To optimize the use of available animals, competitive repopulating strategies will be used to allow direct intra-animal comparisons of different protocols and vectors. The ability of this Core to provide collaborating investigators with a relevant large animal model should greatly facilitate advances in stem cell biology and the eventual application of hematopoietic stem cell gene therapy to the clinical setting.