Modulation of protein folding pathways: a new platform technology for molecular medicine.

蛋白质折叠途径的调节：分子医学的新平台技术。

• 批准号: LP0347910
• 负责人: Prof Stephen Bottomley
• 金额: $ 15.71万
• 依托单位: Monash University
• 依托单位国家: 澳大利亚
• 项目类别: Linkage Projects
• 财政年份: 2003
• 资助国家: 澳大利亚
• 起止时间: 2003-07-01 至 2008-12-31
• 项目状态: 已结题
• 来源: https://dataportal.arc.gov.au/RGS/Web/Grants/LP0347910
• 关键词: Modulation; protein; folding; pathways; new

Misfolding of proteins is becoming recognised as a major cause of inherited disease. We propose to develop a chemical agent that will optimise the folding of alpha1-antitrypsin (AAT), misfolding of which gives rise to inheritable liver and lung disease. This agent will have potential application as a therapy for sufferers of AAT-misfolding disease and for improving the yield of AAT purified from human plasma, which is the current agent used to treat patients with AAT-misfolding disease.

蛋白质的错误折叠被认为是遗传性疾病的主要原因。我们建议开发一种化学试剂来优化α1-抗胰蛋白酶(AAT)的折叠，AAT的错误折叠会导致遗传性肝和肺疾病。该制剂具有潜在的应用前景，可用于治疗AAT错折叠病患者和提高目前用于治疗AAT错折叠病患者的人血浆中纯化的AAT产率。