CORE--DNA/RNA ANALYTICAL LABORATORY

该中心将作为中心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有效性，并监测临床试验（项目）的总体进展。这将通过验证干细胞的转导效率;确定这些细胞中治疗基因的活性表达的存在;检测患者样品中转导的逆转录病毒载体的存在和长期表达，以及通过结合项目2中进行的试验评价核酶的抗病毒活性来实现。核心对IPCP计划的重要性。对于科学项目和两项基因治疗试验，本核心将确定关键终点的确定，即转导细胞中是否存在核酶转基因。

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.