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和NHLBI研究人员提供三个主要功能。1)该中心将提供来自非人类灵长类动物，狒狒（Papio cynocephalus sp.）和长尾猕猴（Macaca nemistrina）的造血祖细胞和干细胞群，用于体外基因转移实验。2)将进行从动员血液和/或引物骨髓中纯化的基因修饰的骨髓再生细胞的体内移植实验。3)开展研究，测试用于干细胞基因治疗的新型干细胞/祖细胞动员方案。当研究人员试图将小鼠和其他小动物模型的发现推断到人类临床环境时，获取大型动物移植模型用于造血干细胞基因治疗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。核心将提供给所有PET支持的研究人员以及其他NHLBI研究人员。与细胞获取和处理中心合作，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，核心将利用已经存在于威斯康星大学区域灵长类动物研究中心和FHCRC的狒狒干细胞移植和基因转移的活动。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、转导、动物移植后细胞分析和方案制定方面的专业知识。为了优化现有动物的使用，将采用竞争性繁殖策略，以便对不同的协议和载体进行直接的动物内比较。该核心为合作研究人员提供相关大型动物模型的能力，将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治疗应用于临床。

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.