Core--Vector

病毒载体核心设施是北卡罗来纳大学基因治疗中心的一个组成部分。它已经运行了8年多，并在此期间保持了指数级的产量增长。这一增长是通过迅速整合新技术发展来实现的，因为它们是可用的，并致力于使这些技术的成果尽可能广泛地为研究人员所利用。这些目标的追求将在拟议的研究计划内继续进行。将增加到载体核心的新技术和服务将包括辅助性腺病毒载体（HD-Ad）、嵌合衣壳腺相关病毒载体（chAAV）、双链（或自互补）腺相关病毒载体（dsAAV）、副粘病毒载体的生产，以及大规模质粒制备，以促进实验室规模病毒载体的生产。这些服务将与MTCC研究人员及其囊性纤维化和代谢性疾病研究项目的具体需求和研究兴趣相吻合。其中一些新服务还将在拟议的试点和可行性研究中发挥重要的支持作用，即生产chAAV （McCarty， P.I.）和副粘病毒载体（Pickles， P.I.）。

The Viral Vector Core Facility is an integral component of the UNC Gene Therapy Center. It has been in operation for over 8 years and has maintained an exponential level of production growth over this time. This growth has 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. Among the new technologies and services to be added to the Vector Core will be the production of helper-dependent adenovirus vectors (HD-Ad), chimeric capsid adeno-associated virus vectors (chAAV), double-strand (or self-complementary) adeno-associated virus vectors (dsAAV), paramyxovirus vectors, and large-scale plasmid preparations to facilitate the production of experimental laboratory-scale stocks of viral vectors. Each of these services will dovetail with the specific needs and research interests of the MTCC investigators and their cystic fibrosis and metabolic disease research programs. Some of these new services will also play an important supporting role in the proposed pilot and feasibility studies, i.e., production of chAAV (McCarty, P.I.) and paramyxovirus vectors (Pickles, P.I.).