CLINICAL TRIAL: PYRIMETHAMINE FOR TREATMENT OF SOD1 MEDIATED AMYLOTROPHIC LATERA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 肌萎缩侧索硬化症是一种运动神经元退行性疾病，无法治疗，通常是致命的。一些病例是由编码胞质超氧化物歧化酶(SOD1)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SOD1介导的5例ALS患者SOD1表达的假说。这项研究是基于观察到乙胺嘧啶在体外细胞和ALS转基因小鼠模型中抑制SOD1的表达。

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.