Core C--Viral Vector Core

北卡罗来纳大学的病毒载体核心设施对我们开发囊性纤维化和其他肺部疾病的基因疗法至关重要。自1993年以来，载体核心一直支持UNC囊性纤维化基因治疗(CF)项目，并作为重组病毒载体试剂的可靠和经济有效的来源，在基因治疗界取得了很大的地位。核心例行地提供如果商业购买则无法获得和/或昂贵得令人无法获得的载体。近年来，对这些试剂的需求迅速增长，使我们进入了持续运营的成熟阶段。我们工作人员齐全，设备齐全，并致力于在可预见的未来提供良好的工作空间。这些 取得这些成就的方法是，在新技术发展成熟时迅速采用这些技术，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，载体核心将负责大规模生产和纯化基因改组和表型选择的嵌合rAAV载体，从而促进这些试剂的表征。此外，Core将扩展其rAAV的生产技术，以提高我们更有效和更大规模生产载体的能力。这将需要使用连续诱导的细胞系 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，将在媒介核心中使用新的EIAV生产者细胞系来产生更安全和更具可预测性的慢病毒载体。对于项目3，病媒核心将建立RSV病媒的生产，因为它们是在PPG周期的过程中开发的。通过这些功能，并与本应用中的其他核心一起，载体核心将支持开发安全有效的CF基因疗法的研究。

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.