Department of Gastroenterology and Hepatology, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai Institute of Digestive Disease, Shanghai, 200001, China.
Division of Gastroenterology and Hepatology, Key Laboratory of Gastroenterology and Hepatology, Ministry of Health, State Key Laboratory for Oncogenes and Related Genes, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai Institute of Digestive Disease, 145 Middle Shandong Road, Shanghai, 200001, China.
Adv Exp Med Biol. 2020;1253:259-283. doi: 10.1007/978-981-15-3449-2_10.
Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease with non-suppurative destruction of the intrahepatic bile ducts. The interplay of genetics and environmental triggers contributes to the onset of the disease and subsequently results in cholestasis and progressive fibrosis. Recently, genome-wide association studies (GWAS) have identified multiple genes influencing the susceptibility to PBC in HLA and non-HLA loci. However, it is estimated that the known risk variants merely account for no more than 20% of the heritability of PBC and causes of the remaining heritability remain uncertain. Increasing evidence suggests that the presence of epigenetic abnormalities may explain the "missing heritability" that cannot be captured by GWAS. Among these epigenetic mechanisms, DNA methylation, histone modification, and noncoding RNAs (i.e. miRNA and lncRNA) are involved in the pathogenesis of PBC. Additionally, telomere dysregulation in biliary epithelial cells (BECs) may play a role in disease onset, whereas a deficiency in sex chromosome and skewed gene expression in the X chromosome may to some extent explain the female dominance in PBC.
原发性胆汁性胆管炎(PBC)是一种慢性胆汁淤积性肝病,伴有肝内胆管非化脓性破坏。遗传和环境触发因素的相互作用导致疾病的发生,随后导致胆汁淤积和进行性纤维化。最近,全基因组关联研究(GWAS)已经确定了多个影响 PBC 在 HLA 和非 HLA 基因座易感性的基因。然而,据估计,已知的风险变异仅占 PBC 遗传率的 20% 以下,其余遗传率的原因仍不确定。越来越多的证据表明,表观遗传异常的存在可能解释了 GWAS 无法捕捉到的“缺失遗传率”。在这些表观遗传机制中,DNA 甲基化、组蛋白修饰和非编码 RNA(即 miRNA 和 lncRNA)参与了 PBC 的发病机制。此外,胆管上皮细胞(BEC)中端粒的失调可能在疾病发病中起作用,而性染色体的缺陷和 X 染色体上基因表达的偏斜在某种程度上解释了 PBC 中女性的优势。