Core C--Viral Vector Core

北卡罗来纳大学病毒载体核心设施对于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，Vector Core一直支持UNC囊性纤维化（CF）基因治疗项目，并作为可靠和具有成本效益的重组病毒载体试剂来源，在基因治疗界取得了很大的地位。核心通常提供无法获得的载体和/或如果在商业上购买则价格昂贵。近年来对这些试剂的需求快速增长，使我们进入了稳定运营的成熟阶段。我们人员配备齐全，设备齐全，并承诺在可预见的未来提供良好的工作空间。这些

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.