Institut Pasteur de Montevideo, Mataojo 2020, Montevideo, CP 11400, Uruguay.
Pediatr Nephrol. 2011 Aug;26(8):1181-95. doi: 10.1007/s00467-010-1697-5. Epub 2010 Nov 27.
Ciliary dysfunction has emerged as a common factor underlying the pathogenesis of both syndromic and isolated kidney cystic disease, an observation that has contributed to the unification of human genetic disorders of the cilium, the ciliopathies. Such grouping is underscored by two major observations: the fact that genes encoding ciliary proteins can contribute causal and modifying mutations across several clinically discrete ciliopathies, and the emerging realization that an understanding of the clinical pathology of one ciliopathy can provide valuable insight into the pathomechanism of renal cyst formation elsewhere in the ciliopathy spectrum. In this review, we discuss and attempt to stratify the different lines of proposed cilia-driven mechanisms for cystogenesis, ranging from mechano- and chemo-sensation, to cell shape and polarization, to the transduction of a variety of signaling cascades. We evaluate both common trends and differences across the models and discuss how each proposed mechanism can contribute to the development of novel therapeutic paradigms.
纤毛功能障碍已成为综合征性和孤立性肾囊性疾病发病机制的共同因素,这一观察结果促进了人类纤毛遗传疾病(纤毛病)的统一。这一分类主要基于两个重要观察结果:一是编码纤毛蛋白的基因可以在几种临床离散的纤毛病中引发因果和修饰性突变,二是人们逐渐认识到,对一种纤毛病的临床病理学的了解可以为纤毛病谱中其他部位肾囊肿形成的病理机制提供有价值的见解。在这篇综述中,我们讨论并试图对不同的纤毛驱动囊肿发生机制进行分层,从机械和化学感觉、细胞形状和极化,到各种信号转导级联的转导。我们评估了模型之间的共同趋势和差异,并讨论了每个提出的机制如何为新的治疗范例的发展做出贡献。