Favreau Catherine, Higuet Dominique, Courvalin Jean-Claude, Buendia Brigitte
Département de Biologie Cellulaire, Institut Jacques Monod, CNRS, Universités Paris 6 & 7, 75251 Paris cedex 05, France.
Mol Cell Biol. 2004 Feb;24(4):1481-92. doi: 10.1128/MCB.24.4.1481-1492.2004.
Autosomal dominantly inherited missense mutations in lamins A and C cause several tissue-specific diseases, including Emery-Dreifuss muscular dystrophy (EDMD) and Dunnigan-type familial partial lipodystrophy (FPLD). Here we analyze myoblast-to-myotube differentiation in C2C12 clones overexpressing lamin A mutated at arginine 453 (R453W), one of the most frequent mutations in EDMD. In contrast with clones expressing wild-type lamin A, these clones differentiate poorly or not at all, do not exit the cell cycle properly, and are extensively committed to apoptosis. These disorders are correlated with low levels of expression of transcription factor myogenin and with the persistence of a large pool of hyperphosphorylated retinoblastoma protein. Since clones mutated at arginine 482 (a site responsible for FPLD) differentiate normally, we conclude that C2C12 clones expressing R453W-mutated lamin A represent a good cellular model to study the pathophysiology of EDMD. Our hypothesis is that lamin A mutated at arginine 453 fails to build a functional scaffold and/or to maintain the chromatin compartmentation required for differentiation of myoblasts into myocytes.
核纤层蛋白A和C的常染色体显性遗传错义突变会引发多种组织特异性疾病,包括埃默里-德赖富斯肌营养不良症(EDMD)和邓尼根型家族性部分脂肪营养不良症(FPLD)。在此,我们分析了在精氨酸453(R453W)处发生突变的核纤层蛋白A过表达的C2C12克隆中的成肌细胞向肌管的分化情况,R453W是EDMD中最常见的突变之一。与表达野生型核纤层蛋白A的克隆不同,这些克隆分化不良或完全不分化,不能正常退出细胞周期,并广泛发生凋亡。这些异常与转录因子肌细胞生成素的低表达水平以及大量高磷酸化视网膜母细胞瘤蛋白的持续存在相关。由于在精氨酸482(一个与FPLD相关的位点)处发生突变的克隆能够正常分化,我们得出结论,表达R453W突变型核纤层蛋白A的C2C12克隆是研究EDMD病理生理学的良好细胞模型。我们的假设是,在精氨酸453处发生突变的核纤层蛋白A无法构建功能性支架和/或维持成肌细胞分化为肌细胞所需的染色质区室化。