CORE--EXPRESSION

基因转移、mRNA和蛋白质表达以及 生物学功效对于基因的成功开发至关重要， 转移载体和药物治疗。一种表达式核心， 在过去的预算中，约翰霍普金斯大学一直在运作 补助金的期限。这个核心在化验方面有相当的专业知识， 通过一般测定，例如： 1. 36 Cl外排和更具体的测定，例如膜片钳; 2.评估基因转移、mRNA表达、载体脱落和扩散 在动物和患者样本中 3.蛋白质表达测定 在上一个预算期间，这一核心的工作发挥了重要作用 在腺病毒和腺相关病毒系统的鉴定中 作为潜在的治疗剂， 有治疗作用的基因转移技术的最新发展， 潜在的治疗和新的药理学治疗， 苯丁酸盐大大增加了对服务的需求 我们的表达核心Expression Core既是一项研究， 开发核心，并为项目提供支持， 研发中心。

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.