Expression Core

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证明对 成功开发基因转移载体和药物治疗。表达式核心具有 自拨款开始以来一直在约翰·霍普金斯大学运作。这个核心有相当大的 具备通过下列方法分析CFTR正常和缺陷功能的专业知识： 1.膜片钳等功能检测； 2.评估基因转移、mRNA表达、载体脱落和在动物和患者中的传播 样本 3.蛋白质表达的分析 在上一个预算期间，这一核心的工作对继续发展 腺相关病毒作为一种非常有前途的治疗剂。基因研究的最新进展 转让技术作为潜在的治疗方法，大大增加了对我们的服务的需求 Expression Core。沿着这些思路，核心在灵长类实验方面发展了相当多的专业知识 在恒河猴和最近的黑猩猩身上。表达核心既是一种研究，也是一种 此外，还为该基因治疗中心内的项目提供支持。 相关性(请参阅说明)：

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 since the beginning of the grant. This Core has considerable expertise in assaying for normal and defective function of CFTR by methods such as: 1. Functional 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 continued development of adeno-associated virus as an extremely promising therapeutic agent. The recent development of gene transfer technologies as potential therapies have increased considerably the demand for the services of our Expression Core. Along these lines, the Core has developed considerable expertise in primate experiments in Rhesus monkeys and more recently in chimpanzees. The Expression Core functions as both a research and development Core, and in addition, provides support for the projects within this Gene Therapy Center. RELEVANCE (See instructions):