CORE--EXPRESSION

基因的蛋白质表达和生物学功效的展示 传递系统对于基因工程的成功开发至关重要， 转移载体和疗法。细胞生理学和组织培养核心 自1989年以来，约翰霍普金斯大学一直在运作， 囊性纤维化基金会通过研究开发计划。 这 核心在以下领域具有相当的专业知识： CFTR的正常和有缺陷的功能，通过一般测定，如：1. 36Cl- 外排和SPQ以及更特异性的测定如膜片钳; 2. 在兔和鸡中产生多克隆抗CFTR抗体; 3. 通过蛋白质印迹分析蛋白质表达;和4.确定 CFTR在异源细胞和动物（兔、大鼠）中的分布 和灵长类动物）细胞。这一核心的工作有助于 腺病毒（12）和腺相关病毒（2，3 -6）鉴定 系统作为潜在的治疗剂， 最近，RAC（重组DNA）批准了五种方案 委员会）。基因转移技术的最新发展， 潜在的治疗方法增加了对我们表达核心的需求。的 目前的细胞生理学和组织培养核心将扩大，以满足 这个新基因治疗中心的需求核心表达将是 既是研究和发展的核心，也是为 在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.