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.
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