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

丙型肝炎感染的基因治疗--慢性传染病模型

• 批准号: 6103644
• 负责人: J W SHIH
• 金额: --
• 依托单位: CLINICAL CENTER
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/6103644
• 关键词: chronic disease /disorder; cytotoxic T lymphocyte; disease /disorder model; epitope mapping; gene expression; gene therapy; hematopoietic stem cells; hepatitis C; hepatitis C virus; laboratory mouse; virus genetics

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.

大约一年前， 一个全面的研究和开发计划，以满足 对未来输血服务的挑战性需求。作为我们 服务责任，我们将被要求提供最先进的 为内部其他部门提供质量支持和咨询 院为了补充我们在病毒方面的专业知识和经验， 肝炎研究，为了有选择性和集中，我们选择 在传染病模型上建立这个新的研发项目到 研究基因治疗作为人类治疗方法 丙型肝炎感染，并开发这种方法作为一种模型， 在慢性传染病方面，我们订立了以下目标：（a） 建立一个功能上可测量的基因表达模型， 使用病毒介导的基因递送系统的造血细胞 丙型肝炎病毒（HCV）基因;（B）开发谱系特异性 HCV基因在造血细胞中的基因表达;（c） 证明谱系特异性基因表达的功效， 用CTL测定和肿瘤杀伤测定进行功能评估 针对HCV特异性表位;和，（d）建立谱系- 特异性基因表达作为治疗慢性 HCV感染。在过去的一年里，在小鼠模型中， 评价了病毒基因传递系统和已建立的CTL测定法 抗HCV核心和包膜蛋白。