CORE--EXPRESSION

基因的蛋白质表达及其生物学效应的论证 递送系统是基因成功开发的关键 转移媒介和治疗。一种细胞生理学和组织培养核心 自1989年以来一直在约翰·霍普金斯大学运作，资金来自 囊性纤维化基金会通过研究发展计划。这 CORE在以下领域拥有相当多的专业知识： 通过常规方法检测CFTR的正常功能和缺陷功能，如：1.36Cl- 外排和SPQ以及膜片钳等更特异的检测方法； 在兔和鸡体内产生多克隆抗CFTR抗体； 蛋白印迹法检测蛋白表达；4.测定 CFTR在异种细胞和动物(兔、大鼠)中的分布 和灵长类)细胞。来自这个核心的工作在 腺病毒(12)和腺相关病毒(2，3-6)的鉴定 系统作为潜在的治疗剂引起了极大的兴奋 最近并导致了RAC(重组DNA)批准的五个方案 委员会)。基因转移技术的最新发展 潜在的治疗方法增加了我们对Expression Core的需求。这个 当前的细胞生理学和组织培养核心将扩展以满足 这个新的基因治疗中心的需求。Expression Core将是 既是研发核心，又为 这个基因治疗中心的项目。

The demonstration of protein expression and biological efficacy of gene delivery systems is critical to the successful development of gene transfer vectors and therapies. A cell physiology and tissue culture core has been in operation at Johns Hopkins University since 1989 funded by the Cystic Fibrosis Foundation through the Research Development Program. This core has considerable expertise in the following areas: assaying for normal and defective function of CFTR by general assays such as: 1. 36Cl- efflux and SPQ and more specific assays such as patch clamping; 2. generating polyclonal anti-CFTR antibodies in both rabbit and chicken; 3. assaying for protein expression by Western blotting; and 4. determining the distribution of CFTR in heterologous cells and in animal (rabbit, rat and primate) cells. Work from this core was instrumental in the identification of adenoviral (12) and adeno-associated virus (2,3-6) systems as potential therapeutic agents which has caused much excitement lately and resulted in five protocols approved by the RAC (Recombinant DNA committee). The recent development of gene transfer technologies as potential therapies has increased the need of our Expression Core. The current Cell Physiology and Tissue Culture Core will be expanded to meet the needs of this new Gene Therapy Center. The Expression Core will be both a research and development core as well as providing support for the projects within this Gene Therapy Center.