Core C--Viral Vector Core

在我们努力开发囊性纤维化和其他肺部疾病的基因治疗中，HIV-Viral Vector Core Facility至关重要。自1993年以来，Vector Core一直支持囊性纤维化（CF）的基因治疗项目，并作为重组病毒载体试剂的可靠和具有成本效益的来源，在基因治疗界取得了很大的地位。核心通常提供商业上无法获得和/或价格过高的载体。近年来，对这些试剂的需求迅速增长，使我们进入了稳定运行的成熟阶段。我们配备了充足的人员，设备齐全，并承诺在可预见的未来提供出色的工作空间。这些 取得这些成就的原因是，在新的技术发展出现时迅速将其纳入，并致力于使研究人员尽可能广泛地获得这些技术的收益。这些目标的追求将在拟议的研究计划中继续进行。在项目1中，载体核心将负责基因改组和表型选择的嵌合rAAV载体的大规模生产和纯化，从而促进这些试剂的表征。此外，核心将扩大其生产技术的rAAV，以提高我们的能力，生产载体更有效和更大规模。这将需要使用连续的细胞系诱导 通过辅助病毒感染，使用哺乳动物以及SF-9昆虫细胞。在项目2中，新的EIAV生产细胞系将用于载体核心，以产生具有更高安全性和可预测性的慢病毒载体。对于项目3，Vector Core将在PPG周期过程中开发RSV载体时开始生产RSV载体。通过这些功能，并与本申请中的其他核心一起，Vector Core将支持CF安全有效基因治疗的研究。

The UNC Viral Vector Core Facility is essential in our effort to develop gene therapy for cystic fibrosis and other pulmonary diseases. The Vector Core has supported the UNC Gene Therapy for Cystic Fibrosis (CF) projects since 1993 and has achieved a great deal of status in the gene therapy community as a reliable and cost-effective source of recombinant virus vector reagents. The core routinely makes available vectors that are unavailable and/or prohibitively expensive if purchased commercially. Rapid growth in demand for these reagents in recent years has brought us to a mature stage of consistent operation. We are fully staffed, wellequipped, and have the benefit of a commitment for excellent working space for the foreseeable future. These achievements have been accomplished by rapid incorporation of new technological developments as they become available, and by a commitment to making the proceeds of these technologies as widely accessible to researchers as possible. The pursuit of these goals will continue within the proposed research program. In Project 1, the Vector Core will be responsible for the large-scale production and purification of genetically shuffled and phenotypically selected chimeric rAAV vectors, thus facilitating the characterization of these reagents. Additionally, the Core will broaden its production techniques for rAAV to increase our ability to produce vectors more efficiently and on a larger scale. This will entail the use of continuous cell lines induced by helper virus infection, using mammalian as well as SF-9 insect cells. In Project 2, new EIAV producer cell lines will be used in the Vector Core to generate lentivirus vectors with greater safety and predictability. For Project 3, the Vector Core will institute production of RSV vectors as they are developed over the course of the PPG cycle. Through these functions, and in conjunction with other Cores in this application, the Vector Core will support research towards the development of safe and effective gene therapy for CF.