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于 成功开发基因转移载体和药物治疗。表达式核心具有 自赠款开始以来，该项目一直在约翰霍普金斯大学运作。这一核心具有相当大的 通过以下方法检测CFTR的正常和缺陷功能的专业知识： 1.功能测定，如膜片钳; 2.评估动物和患者中的基因转移、mRNA表达、载体脱落和扩散 样品 3.蛋白质表达测定 在上一个预算期间，该核心的工作有助于继续发展 腺相关病毒作为一个非常有前途的治疗剂。基因工程的最新进展 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。 表达核心。沿着这些路线，内核在灵长类动物实验方面已经发展出相当多的专业知识 在恒河猴和最近的黑猩猩身上。Expression Core既是一项研究， 和发展核心，并提供支持的项目在这个基因治疗中心。 相关性（参见说明）：