Laboratory of Muscle Research and Mitochondrial Function-CELLEX, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Faculty of Medicine and Health Sciences, University of Barcelona (UB), Department of Internal Medicine-Hospital Clínic of Barcelona, Barcelona, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Madrid, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CIBERNED), Instituto de Salud Carlos III, Madrid, Spain; Universitat Pompeu Fabra (UPF), Barcelona, Spain.
Neurobiol Aging. 2018 May;65:206-216. doi: 10.1016/j.neurobiolaging.2018.01.021. Epub 2018 Feb 7.
Mutations in the parkin gene (PRKN) are the most common cause of autosomal-recessive juvenile Parkinson's disease (PD). PRKN encodes an E3 ubiquitin ligase that is involved in multiple regulatory functions including proteasomal-mediated protein turnover, mitochondrial function, mitophagy, and cell survival. However, the precise molecular events mediated by PRKN mutations in PRKN-associated PD (PRKN-PD) remain unknown. To elucidate the cellular impact of parkin mutations, we performed an RNA sequencing study in skin fibroblasts from PRKN-PD patients carrying different PRKN mutations (n = 4) and genetically unrelated healthy subjects (n = 4). We identified 343 differentially expressed genes in PRKN-PD fibroblasts. Gene ontology and canonical pathway analysis revealed enrichment of differentially expressed genes in processes such as cell adhesion, cell growth, and amino acid and folate metabolism among others. Our findings indicate that PRKN mutations are associated with large global gene expression changes as observed in fibroblasts from PRKN-PD patients and support the view of PD as a systemic disease affecting also non-neural peripheral tissues such as the skin.
PRKN 基因突变是常染色体隐性遗传青少年帕金森病(PD)最常见的原因。PRKN 基因编码一种 E3 泛素连接酶,参与多种调节功能,包括蛋白酶体介导的蛋白质降解、线粒体功能、线粒体自噬和细胞存活。然而,PRKN 相关 PD(PRKN-PD)中 PRKN 突变介导的确切分子事件仍不清楚。为了阐明 parkin 突变的细胞影响,我们对来自携带不同 PRKN 突变的 PRKN-PD 患者(n=4)和遗传上无关的健康受试者(n=4)的皮肤成纤维细胞进行了 RNA 测序研究。我们在 PRKN-PD 成纤维细胞中鉴定出 343 个差异表达基因。基因本体论和经典途径分析显示,差异表达基因在细胞黏附、细胞生长以及氨基酸和叶酸代谢等过程中富集。我们的研究结果表明,PRKN 突变与 PRKN-PD 患者成纤维细胞中观察到的大规模基因表达变化有关,并支持 PD 是一种全身性疾病的观点,也影响非神经外周组织,如皮肤。