CORE--EXPRESSION

基因转移、信使核糖核酸和蛋白质表达及 生物效应是基因成功发育的关键 转移载体和药物治疗。表达式核心具有 在过去的预算期间一直在约翰·霍普金斯大学运作 授权期。这个核心在化验方面有相当多的专业知识 对于CFTR的正常和有缺陷的功能，通过一般分析，例如： 1.36Cl-外排和更特异的检测方法，如膜片钳； 2.评估基因转移、mrna表达、载体脱落和传播。 在动物和患者样本中 3.蛋白质表达的分析 在前一个预算期间，这一核心工作起到了重要作用 在鉴定腺病毒和腺相关病毒系统中的应用 作为潜在的治疗剂，以及苯丁酸酯作为潜在的 有治疗作用。基因转移技术的最新发展 潜在的疗法和新的药物疗法，如 丁酸苯酯大大增加了对服务的需求 我们的表情核心。《表达核心》既是一项研究，也是一项 开发核心，并为该项目提供支持 研究发展中心。

The demonstration of gene transfer, mRNA and protein expression and biological efficacy is critical to the successful development of gene transfer vectors and pharmacologic therapies. An expression core has been in operation at John Hopkins University during the past budget period of the grant. This core has considerable expertise in assaying for normal and defective function of CFTR by general assays such as: 1. 36Cl-efflux and more specific assays such as patch clamping; 2. evaluating gene transfer, mRNA expression, vector shedding and spread in animal and patients samples 3. assays for protein expression Work from this core during the previous budget period was instrumental in the identification of adenoviral and adeno-associated virus systems as potential therapeutic agents and also phenylbutyrate as a potential therapeutic. The recent development of gene transfer technologies as potential therapies and new pharmacologic therapies such as phenylbutyrate have increased considerably the demand for the services our Expression Core. The Expression Core is both a research and development core and provides support for the projects within this Research Development Center.