GENE THERAPY FOR HEPATITIS C INFECTION--CHRONIC INFECTIOUS DISEASE MODEL

大约一年前，一项新的倡议开始制定一项全面的 研发计划，以满足未来的挑战性需求 输血服务作为我们服务职责的一部分，我们将 需要提供最先进的质量支持和咨询， 研究所内的其他部门。为了补充我们的专业知识， 有病毒性肝炎研究的经验，要有选择性和重点， 我们选择在传染病模型上建立这个新的研发项目。到 丙型肝炎基因治疗研究 感染，并开发这种方法作为慢性感染的模型， 为预防疾病，我们制定了以下目标：（a）建立一个 造血细胞中功能可测量的基因表达模型， 丙型肝炎病毒（HCV）的病毒介导的基因递送系统 （B）在造血细胞中发展谱系特异性基因表达 细胞的HCV基因;（c）以证明谱系特异性 通过CTL试验和肿瘤杀伤功能评估的基因表达 针对HCV特异性表位的测定;以及（d）建立谱系- 特异性基因表达作为抗慢性HCV治疗方式 感染在过去的一年里，在小鼠模型中，我们评估了一种病毒， 基因递送系统和针对HCV核心的已建立的CTL测定， 包膜蛋白

About a year ago a new initiative started to develop a comprehensive research and development program to meet the challenging demand of future transfusion services. As part of our service responsibility, we will be required to provide state-of-the-art quality supports and consultation to the other sectors within the institute. To complement our expertise and experience in viral hepatitis research, and to be selective and focused, we chose to build this new R&D program on an infectious disease model. To investigate gene therapy as a therapeutic approach for human hepatitis C infection and to develop this approach as a model for chronic infectious diseases, we have set up the following goals: (a) to establish a functionally measurable gene expression model in hematopoietic cells using a virally-mediated gene delivery system for the hepatitis C virus (HCV) gene; (b) to develop lineage-specific gene expression in hematopoietic cells for HCV genes; (c) to demonstrate the efficacy of lineage-specific gene expression by functional assessment with CTL assays and tumor-killing assays directed to HCV-specific epitopes; and, (d) to establish lineage- specific gene expression as a therapeutic modality against chronic HCV infection. In the past year, in a mouse model, we have evaluated a viral gene delivery system and established CTL assays against HCV core and envelope proteins.