Core--Expression

基因转移、信使核糖核酸和蛋白质的表达以及生物学效应的验证是基因转移载体和药物治疗成功的关键。在过去的赠款预算期间，表达核心一直在约翰霍普金斯大学运行。该核心在通过以下方法分析CFTR的正常和有缺陷的功能方面拥有丰富的专业知识： 1.[36]氯-外流和更特异的检测方法，如膜片钳； 2.评估基因转移、基因表达、载体在动物和患者样本中的脱落和传播； 3.蛋白质表达分析。 在上一个预算期间，该核心的工作有助于确定腺病毒和腺相关病毒系统为潜在的治疗剂，并将苯丁酸酯确定为潜在的治疗剂。基因转移技术的最新发展，作为潜在的治疗方法和新的药物治疗，如苯丁酸酯，极大地增加了对我们的Expression Core服务的需求。表达核心既是研究和开发的核心，又为该基因治疗中心内的项目提供支持。

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 Johns 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 methods such as: 1. [36]Cl- 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. assaying 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 of our Expression Core. The Expression Core functions as both a research and development Core, and in addition provides support for the projects within this Gene Therapy Center.