CORE--DNA/RNA ANALYTICAL LABORATORY

该中心将作为中央PCR诊断实验室，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）的整体进展。这将通过验证干细胞的转导效率来完成；在这些细胞中建立治疗基因的活性表达；检测患者样本中转导逆转录病毒载体的存在和长期表达，并结合项目2中的试验评估核酶的抗病毒活性。核心对IPCP计划的重要性。关键终点的确定，即核酶转基因在转导细胞中的存在，将由该核心为科学项目和两个基因治疗试验确定。

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.