CLINICAL TRIAL: PYRIMETHAMINE FOR TREATMENT OF SOD1 MEDIATED AMYLOTROPHIC LATERA

临床试验：乙胺嘧啶治疗 SOD1 介导的肌萎缩侧索硬化症

• 批准号: 7731272
• 负责人: Robert H. Brown
• 金额: $ 0.16万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-04-01 至 2008-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7731272
• 关键词: Amyotrophic Lateral Sclerosis; Cells; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Degenerative Disorder; Depressed mood; Funding; Genes; Grant; In Vitro; Institution; Mediating; Motor Neurons; Mutation; Patients; Pharmaceutical Preparations; Purpose; Pyrimethamine; Research; Research Personnel; Resources; Source; Superoxide Dismutase; Testing; Transgenic Organisms; United States National Institutes of Health; mouse model

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Amyotrophic lateral sclerosis is a degenerative disorder of motor neurons that is untreatable and uniformly fatal. Some cases are caused by mutations in the gene encoding cytosolic superoxide dismutase (SOD1). The purpose of this study is to test the hypothesis that the drug pyrimethamine can inhibit the expression of SOD1 in five patients with SOD1-mediated ALS. This study is predicated on the observation that pyrimethamine depresses SOD1 expression in cells in vitro and in a transgenic mouse model of ALS.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 肌萎缩性侧索硬化症是一种无法治愈且致命的运动神经元退行性疾病。 一些病例是由编码细胞溶质超氧化物歧化酶（SOD 1）的基因突变引起的。 本研究的目的是验证药物乙胺嘧啶可以抑制SOD 1介导的ALS患者的SOD 1表达的假设。 这项研究是预测的观察，乙胺嘧啶抑制SOD 1在体外细胞和转基因小鼠模型的ALS的表达。