Kaganovich Daniel, Kopito Ron, Frydman Judith
Department of Biology and BioX Program, Stanford University, Stanford, California 94305, USA.
Nature. 2008 Aug 28;454(7208):1088-95. doi: 10.1038/nature07195.
The accumulation of misfolded proteins in intracellular amyloid inclusions, typical of many neurodegenerative disorders including Huntington's and prion disease, is thought to occur after failure of the cellular protein quality control mechanisms. Here we examine the formation of misfolded protein inclusions in the eukaryotic cytosol of yeast and mammalian cell culture models. We identify two intracellular compartments for the sequestration of misfolded cytosolic proteins. Partition of quality control substrates to either compartment seems to depend on their ubiquitination status and aggregation state. Soluble ubiquitinated misfolded proteins accumulate in a juxtanuclear compartment where proteasomes are concentrated. In contrast, terminally aggregated proteins are sequestered in a perivacuolar inclusion. Notably, disease-associated Huntingtin and prion proteins are preferentially directed to the perivacuolar compartment. Enhancing ubiquitination of a prion protein suffices to promote its delivery to the juxtanuclear inclusion. Our findings provide a framework for understanding the preferential accumulation of amyloidogenic proteins in inclusions linked to human disease.
细胞内淀粉样蛋白包涵体中错误折叠蛋白的积累是许多神经退行性疾病(包括亨廷顿舞蹈症和朊病毒病)的典型特征,据认为这是在细胞蛋白质质量控制机制失效后发生的。在此,我们研究了酵母和哺乳动物细胞培养模型的真核细胞质中错误折叠蛋白包涵体的形成。我们确定了两个用于隔离错误折叠的胞质蛋白的细胞内区室。质量控制底物分配到任一区室似乎取决于它们的泛素化状态和聚集状态。可溶性泛素化错误折叠蛋白积聚在蛋白酶体集中的近核区室中。相反,终末聚集蛋白被隔离在液泡周围包涵体中。值得注意的是,与疾病相关的亨廷顿蛋白和朊病毒蛋白优先被导向液泡周围区室。增强朊病毒蛋白的泛素化足以促进其转运至近核包涵体。我们的研究结果为理解淀粉样蛋白生成蛋白在与人类疾病相关的包涵体中的优先积累提供了一个框架。