CORE--DNA/RNA ANALYTICAL LABORATORY

核心--DNA/RNA分析实验室

• 批准号: 6334886
• 负责人: DANIELA CASTANOTTO
• 金额: $ 23.78万
• 依托单位: CITY OF HOPE NATIONAL MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-08-01 至 2001-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6334886
• 关键词: AIDS; AIDS therapy; DNA; RNA; biomedical facility; clinical research; gene therapy; genetic registry /resource /referral center; human tissue; polymerase chain reaction

This Core will serve as the central PCR diagnostic laboratory to evaluate the efficacy of the different strategies implemented in the scientific projects (Projects 1-3) and to monitor the overall progress of the clinical trial (Project). This will be accomplished by verifying the transduction efficiency of stem cells; establishing the presence of an active expression of the therapeutic gene in these cells; detecting the presence and long- term expression of the transduced retroviral vectors in patient samples, and by evaluating the antiviral activity of the ribozyme in conjunction with tests done in Project 2. Importance of Core to IPCP Program. The critical endpoint determination, namely the presence of ribozyme transgene in transduced cells, will be determined by this Core for the scientific projects and for the two gene therapy trials.

该核心将作为中央PCR诊断实验室，以评估科学项目（项目1-3）中实施的不同策略的功效，并监视临床试验（项目）的整体进度。这将通过验证干细胞的转导效率来实现。在这些细胞中建立治疗基因的主动表达。检测到患者样品中转导的逆转录病毒载体的存在和长期表达，并通过评估核酶与项目2中的测试结合进行核酶的抗病毒活性。核心对IPCP计划的重要性。临界端点确定，即转导细胞中核酶转基因的存在，将由该核心在科学项目和两个基因治疗试验中确定。