Expression Core

表达核心

• 批准号: 7669757
• 负责人: William B. Guggino
• 金额: $ 19.39万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-04-01 至 2014-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7669757
• 关键词: Animals; Biological; Biological Assay; Budgets; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Dependovirus; Development; Gene Transfer; Genes; Grant; Instruction; Macaca mulatta; Messenger RNA; Methods; Operative Surgical Procedures; Pan Genus; Patients; Primates; Sampling; Services; Technology; Therapeutic Agents; Universities; Work; expression vector; gene therapy; gene transfer vector; mRNA Expression; patch clamp; protein expression; research and development; research study

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):

基因转移，mRNA和蛋白质表达以及生物学功效的证明对 基因转移载体和药理学疗法的成功开发。表达核心具有 自赠款开始以来，约翰·霍普金斯大学就一直在运营。这个核心有很大的 通过诸如： 1。功能分析，例如贴片夹； 2。评估基因转移，mRNA表达，载体脱落和扩散在动物和患者中 样品 3。分析蛋白质表达 在上一个预算期间，该核心的工作有助于持续发展 腺相关病毒是一种极有前途的治疗剂。基因的最新发展 随着潜在疗法的转移技术已大大增加了对我们服务的需求 表达核心。沿着这些线，核心在灵长类动物实验中发展了丰富的专业知识 在恒河猴和最近的黑猩猩中。表达核心既是一项研究 和开发核心，此外，还为该基因治疗中心内的项目提供了支持。 相关性（请参阅说明）：