CORE--VECTOR PRODUCTION

Vector Core的总体功能是提供高质量的 逆转录病毒载体和腺相关病毒（AAV）载体， 这份SCOR基金的赞助人。几乎所有的项目在这方面 SCOR提案涉及利用逆转录病毒载体作为实验工具 将特定基因导入造血干细胞或 不同的造血细胞系。矢量核心将提供不同的 骨架载体和包装细胞系以及适当的技术 关于工程化高滴度载体的最有效方法的建议 这些不同的研究。Vector Core还将提供 项目6中用于狒狒研究的逆转录病毒载体数量， 将确保这些不同载体具有相似滴度。为 在项目7中提出的人类研究中，载体核心将确保生产 临床级逆转录病毒载体用于基因治疗 白细胞粘附缺陷（LAD）。符合FDA要求的质量控制检测 和RAC要求，包括复制能力病毒的测定， 外源感染因子和逆转录病毒基因组重排将 主要在Vector Core设施中进行，或通过 微生物协会。

The overall function of the Vector Core is to provide high quality retroviral vectors and adeno-associated virus (AAV) vectors to investigatOrs of this SCOR grant. Virtually all of the projects in this SCOR proposal involve utilizing retroviral vectors as an experimental tool to introduce specific genes into hematopoietic stem cells or into different hematopoietic cell lines. The Vector Core will provide different backbone vectors and packaging cell lines as well as appropriate technical advice relating to the most efficient way to engineer high titer vectors for these different studies. The Vector Core will also provide large quantities of retroviral vectors for the baboon studies in Project 6 and will assure that these different vectors are of similar titer. For the human studies proposed in Project 7 the Vector Core will assure production of clinical grade retroviral vectors for use in the gene therapy of leukocyte adherence deficiency (LAD). Quality control assays meeting FDA and RAC requirements including assays for replication-competent virus, adventitious infectious agents, and retroviral genomic rearrangements will largely be carried out in the Vector Core facility or contracted through Microbiological Associates.