MOUSE MODEL FOR DISEASES OF PROTEIN MISFOLDING

蛋白质错误折叠疾病的小鼠模型

• 批准号: 8357844
• 负责人: P. MICHAEL CONN
• 金额: $ 5.82万
• 依托单位: OREGON HEALTH & SCIENCE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-05-01 至 2012-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8357844
• 关键词: Affect; Alzheimer' s Disease; Biological Models; Cataract; Cell Culture Techniques; Cystic Fibrosis; Data; Digestive System Disorders; Disease; Enzymes; Funding; Grant; Huntington Disease; Klinefelter' s Syndrome; Laboratories; Malignant Neoplasms; Modeling; Mutation; National Center for Research Resources; Nephrogenic Diabetes Insipidus; Neurodegenerative Disorders; Parkinson Disease; Persons; Primates; Principal Investigator; Protein-Folding Disease; Proteins; Research; Research Infrastructure; Resources; Retinitis Pigmentosa; Rodent; Source; System; Translating; Translations; United States National Institutes of Health; cost; design; human disease; hypercholesterolemia; in vivo; loved ones; mouse model; protein folding; protein misfolding; prototype

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. This project is designed to develop, characterize, compare and contrast two prototypes for a genetically modified mouse model for a human disease of protein folding. Diseases caused by misfolding include cystic fibrosis, hypogonadotropic hypogonadism, nephrogenic diabetes insipidus, retinitis pigmentosa, hypercholesterolemia, cataracts, neurodegenerative diseases (Huntington's, Alzheimer's and Parkinson's), particular cancers and a number of digestive disorders resulting from enzyme mutation. It is fair to say that virtually every person will be affected by protein folding diseases during his or her lifetime, either directly or due to the illness of a loved one. In spite of this, there are few model systems, and none in rodents, that allow the translation of the available cell-culture data on protein rescue into in vivo systems. A convenient laboratory model for the use of pharmacoperones will be designed in order to translate pharmacoperone use to treatment of human disease.

该副本是利用资源的众多研究子项目之一 由NIH/NCRR资助的中心赠款提供。对该子弹的主要支持 而且，副投影的主要研究员可能是其他来源提供的 包括其他NIH来源。 列出的总费用可能 代表subproject使用的中心基础架构的估计量， NCRR赠款不直接向子弹或副本人员提供的直接资金。 该项目旨在为蛋白质折叠的人类疾病开发，表征，比较和对比两种原型。由误折折叠引起的疾病包括囊性纤维化，性肾上腺素性性腺功能不全，肾脏糖尿病性肠道疾病，色素性视网膜炎，高胆固醇血症，白内障，神经退行性疾病（Huntington，Alzheimer's and Alzheimer's和Parkinson's），特定的犬类和数字疾病，疾病和数字是数字。突变。可以公平地说，几乎每个人都会直接或由于亲人的疾病而受到蛋白质折叠疾病的影响。尽管如此，很少有模型系统，而在啮齿动物中也没有，可以将有关蛋白质救援的可用细胞培养数据转换为体内系统。将设计用于使用药物蛋白酶的方便的实验室模型，以将药物使用量转化为治疗人类疾病。