Moriuchi Takanobu, Muraoka Takuya, Mio Kazuhiro, Osumi Takashi, Hirose Fumiko
Graduate School of Life Science, University of Hyogo, Koto, Kamigori, Hyogo, 678-1297, Japan.
Genes Cells. 2014 Dec;19(12):901-18. doi: 10.1111/gtc.12189. Epub 2014 Oct 16.
Mutation of the lamin A gene (LMNA) causes a diverse range of diseases referred to as laminopathies. Because most laminopathies have a dominant inheritance pattern and progress gradually, cultured cells stably expressing mutant lamin A at the same level as endogenous wild-type cells are required for chronological analysis. In this study, we showed that an expression system involving a lentiviral vector that carries the human metallothionein gene basal promoter ensures stable and basal-level expression of proteins and is thus suitable for investigating the properties of lamin A mutants. The small ubiquitin-related modifier (SUMO) modification (SUMOylation)-defective E203G mutant that is associated with familial dilated cardiomyopathy exhibited abnormal subnuclear distribution and inhibited normal localization of WT lamin A in a dominant-negative manner. Low-level and long-term expression of the E203G mutant resulted in multinucleated giant cells, aberrant lipid droplet accumulation in the cytoplasm and premature senescence. Expression of another SUMOylation-defective mutant (K201R) did not induce any phenotypes observed in cells expressing E203G. These results indicate that the E203G mutant may inhibit the normal functions of wild-type lamin A in a dominant-negative manner, but a defect in SUMOylation itself may not be involved in disease pathogenesis.
核纤层蛋白A基因(LMNA)的突变会引发多种被称为核纤层蛋白病的疾病。由于大多数核纤层蛋白病具有显性遗传模式且病情逐渐发展,因此进行时序分析需要培养出能稳定表达与内源性野生型细胞水平相同的突变型核纤层蛋白A的细胞。在本研究中,我们发现一种涉及携带人金属硫蛋白基因基础启动子的慢病毒载体的表达系统,可确保蛋白质的稳定和基础水平表达,因此适用于研究核纤层蛋白A突变体的特性。与家族性扩张型心肌病相关的小泛素相关修饰物(SUMO)修饰(SUMO化)缺陷型E203G突变体表现出异常的核内亚分布,并以显性负性方式抑制野生型核纤层蛋白A的正常定位。E203G突变体的低水平和长期表达导致多核巨细胞形成、细胞质中异常脂滴积累以及过早衰老。另一种SUMO化缺陷型突变体(K201R)的表达未诱导出在表达E203G的细胞中观察到的任何表型。这些结果表明,E203G突变体可能以显性负性方式抑制野生型核纤层蛋白A的正常功能,但SUMO化本身的缺陷可能与疾病发病机制无关。