Centro de Investigación Príncipe Felipe, Valencia, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Valencia, Spain.
FEBS J. 2018 Jun;285(11):2071-2090. doi: 10.1111/febs.14468. Epub 2018 Apr 23.
Lafora disease (LD) is a fatal neurodegenerative disorder caused mostly by mutations in either of two genes encoding laforin and malin. LD is characterized by accumulation of a poorly branched form of glycogen in the cytoplasm of neurons and other cells. We previously reported dysfunctional mitochondria in different LD models. Now, using mitochondrial uncouplers and respiratory chain inhibitors, we have investigated with human fibroblasts a possible alteration in the selective degradation of damaged mitochondria (mitophagy) in LD. By flow cytometry of MitoTracker-labelled cells and measuring the levels of various mitochondrial proteins by western blot, we found in LD fibroblasts a partial impairment in the increased mitochondrial degradation produced by these treatments. In addition, colocalization of mitochondrial and lysosomal markers decreased in LD fibroblasts. All these results are consistent with a partial impairment in the induced autophagic degradation of dysfunctional mitochondria in LD fibroblasts. However, canonical recruitment of Parkin to mitochondria under these conditions remained unaffected in LD fibroblasts, and also in SH-SY5Y cells after malin and laforin overexpression. Neither mitochondrial localization nor protein levels of Bcl-2-like protein 13, another component of the mitophagic machinery that operates under these conditions, were affected in LD fibroblasts. In contrast, although these treatments raised autophagy in both control and LD fibroblasts, this enhanced autophagy was clearly lower in the latter cells. Therefore, the autophagic degradation of altered mitochondria is impaired in LD, which is due to a partial defect in the autophagic response and not in the canonical mitophagy signalling pathways.
拉佛拉病(LD)是一种致命的神经退行性疾病,主要由编码 laforin 和 malin 的两种基因中的突变引起。LD 的特征是神经元和其他细胞的细胞质中积累了一种分支不良的糖原形式。我们之前在不同的 LD 模型中报告了功能失调的线粒体。现在,我们使用线粒体解偶联剂和呼吸链抑制剂,用人类成纤维细胞研究了 LD 中受损线粒体(自噬体)选择性降解(自噬)的可能改变。通过用 MitoTracker 标记细胞的流式细胞术和通过 Western blot 测量各种线粒体蛋白的水平,我们发现 LD 成纤维细胞中的这些处理引起的线粒体降解增加存在部分损害。此外,LD 成纤维细胞中线粒体和溶酶体标记物的共定位减少。所有这些结果都与 LD 成纤维细胞中功能失调的线粒体诱导自噬降解的部分损害一致。然而,在这些条件下,Parkin 对线粒体的经典募集在 LD 成纤维细胞中不受影响,在过表达 malin 和 laforin 后,SH-SY5Y 细胞也是如此。在 LD 成纤维细胞中,线粒体定位或另一种在这些条件下起作用的自噬体形成机制的 Bcl-2 样蛋白 13 的蛋白水平均不受影响。相比之下,尽管这些处理均增加了对照和成纤维细胞中的自噬,但在后一种细胞中,这种增强的自噬明显较低。因此,LD 中改变的线粒体的自噬体降解受损,这是由于自噬反应的部分缺陷,而不是经典的自噬体形成信号通路。