Development of therapeutic copper delivery agents for Menkes disease

门克斯病治疗性铜输送剂的开发

• 批准号: nhmrc : GNT1113593
• 负责人: Prof Ashley Bush
• 金额: $ 65.15万
• 依托单位国家: 澳大利亚
• 项目类别: Development Grants
• 财政年份: 2016
• 资助国家: 澳大利亚
• 起止时间: 2016-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/development-therapeutic-copper-menkes-disease/1346859
• 关键词: Development; therapeutic; copper; delivery; agents

Menkes disease does not currently have an effective treatment. The disease is caused by genetic defects that reduce copper transport into the brain and cause mental retardation and death. We have developed drugs that deliver copper into the brain and should cure Menkes disease. We aim to demonstrate that our drugs are effective in mice that have the same genetic defect as patients. Successful results will allow us to begin treating Menkes disease patients to determine if we can cure the disease.

门克斯病目前还没有有效的治疗方法。这种疾病是由基因缺陷引起的，这种缺陷减少了铜进入大脑的运输，并导致智力低下和死亡。我们已经开发出将铜输送到大脑中的药物，应该可以治愈门克斯病。我们的目标是证明我们的药物对与患者具有相同基因缺陷的小鼠有效。成功的结果将使我们能够开始治疗门克斯病患者，以确定我们是否可以治愈这种疾病。