Department of Dermatology, Hunan Children's Hospital, Changsha, China.
Department of Dermatology, The Second Xiangya Hospital, Central South University, Changsha, China.
Adv Exp Med Biol. 2020;1253:375-403. doi: 10.1007/978-981-15-3449-2_13.
Scleroderma (systemic sclerosis; SSc) is a complex and highly heterogeneous multisystem rheumatic disease characterized by vascular abnormality, immunologic derangement, and excessive deposition of extracellular matrix (ECM) proteins. To date, the etiology of this life-threatening disorder remains not fully clear. More and more studies show epigenetic modifications play a vital role. The aberrant epigenetic status of certain molecules such as Fli-1, BMPRII, NRP1, CD70, CD40L, CD11A, FOXP3, KLF5, DKK1, SFRP1, and so on contributes to the pathogenesis of progressive vasculopathy, autoimmune dysfunction, and tissue fibrosis in SSc. Meanwhile, numerous miRNAs including miR-21, miR-29a, miR-196a, miR-202-3p, miR-150, miR-let-7a, and others are involved in the process. In addition, the abnormal epigenetic biomarker levels of CD11a, Foxp3, HDAC2, miR-30b, miR-142-3p, miR-150, miR-5196 in SSc are closely correlated with disease severity. In this chapter, we not only review new advancements on the epigenetic mechanisms involved in the pathogenesis of SSc and potential epigenetic biomarkers, but also discuss the therapeutic potential of epigenetic targeting therapeutics such as DNA methylation inhibitors, histone acetylase inhibitors, and miRNA replacement.
硬皮病(系统性硬化症;SSc)是一种复杂且高度异质的多系统风湿性疾病,其特征为血管异常、免疫功能紊乱和细胞外基质(ECM)蛋白过度沉积。迄今为止,这种危及生命的疾病的病因仍不完全清楚。越来越多的研究表明表观遗传修饰起着至关重要的作用。某些分子如 Fli-1、BMPRII、NRP1、CD70、CD40L、CD11A、FOXP3、KLF5、DKK1、SFRP1 等的异常表观遗传状态导致 SSc 中进行性血管病变、自身免疫功能障碍和组织纤维化的发病机制。同时,许多 miRNA 包括 miR-21、miR-29a、miR-196a、miR-202-3p、miR-150、miR-let-7a 等也参与其中。此外,SSc 中 CD11a、Foxp3、HDAC2、miR-30b、miR-142-3p、miR-150、miR-5196 的异常表观遗传生物标志物水平与疾病严重程度密切相关。在本章中,我们不仅回顾了参与 SSc 发病机制的表观遗传机制和潜在的表观遗传生物标志物的新进展,还讨论了表观遗传靶向治疗的治疗潜力,如 DNA 甲基化抑制剂、组蛋白乙酰化酶抑制剂和 miRNA 替代物。