CORE--CELL PROCUREMENT AND PROCESSING RESOURCE

细胞采购和加工资源。细胞采购和处理核心将提供资源，以帮助规避阻碍体外基因治疗领域进展的关键限制，即无法获得实验所需的足够质量的纯化干细胞或其他细胞群体，以及有效基因转导所需条件的优化。此核心将通过提供两个特定功能来解决这一限制。(1)核心将向PEGT研究人员以及其他NHLBI研究人员提供大量浓缩的干细胞或其他特定血细胞群，用于他们的体外研究、灵长类动物模型测试和临床前试验使用。处理核心将从灵长类核心获得样本，并将运行捐赠者招募计划、身体骨髓计划和冷冻保存研究样本储存库，以提供大规模细胞选择所需的细胞群体。(2)利用FHCRC低温生物学实验室提供的仪器和专业知识，该中心将提供临床级别的细胞处理以及从人类和灵长类动物成分中进行选择。核心还将通过执行2色和3色流式细胞仪测定以及定量的氟氯化碳和LTC-IC分析来表征这些浓缩和/或转导的细胞群。这些诊断服务将使研究人员能够对细胞选择的结果进行质量控制，并确定各种干细胞/祖细胞间的转导效率。该中心能够获得、处理、分离和鉴定大量的纯化细胞，并将这些细胞交付给NHLBI研究人员，这将大大加强基础干细胞生物学领域的研究，并极大地促进基于基因治疗的新型应用的临床进展。

Cell procurement and processing resource. The Cell Procurement and processing Core will supply resources to help circumvent a critical limitation that impedes progress in the field of ex-vivo gene therapy, an inability to obtain sufficient qualities of purified stem cells or other cell populations needed for experimentation and optimization of the conditions required for efficient gene transduction. This Core will address that limitation by providing two specific functions. (1) The Core will make available to PEGT researchers as well as other NHLBI investigators large quantities of enriched stem cells or other specific blood cell populations for their in vitro studies, for primate model testing, and for pre-clinical trial usage. The Processing Core will obtain specimens form the Primate Core, and will operate a donor recruitment program, a cadaveric bone marrow program, and a Repository of Cryopreserved Research Specimens to supply the cell populations needed for large-scale cell selections. (2) Taking advantage of the instrumentation and expertise available from within the FHCRC Cryobiology Laboratory, the Core will provide clinical-grade cell processing and selections from both the human and primate components. The Core will also characterize these enriched and/or transduced cell populations by performing 2 and 3 color flow cytometric determinations, and quantitative CFC and LTC-IC assays. These diagnostic services will provide investigators with the ability to quality control results from the cell selections, and to determine efficiencies of transduction in the various stem/progenitor cell compartments. The ability of this Core to obtain, process, isolate and characterize large numbers of purified cells, and to deliver those cells to NHLBI researchers, will significantly enhanced studies in the areas of basic stem cell biology, and greatly facilitate the clinical advancement of novel gene therapy-based applications.