SUMOylation Modulates CFTR Biogenesis: Is the Pathway Druggable?
SUMOylation Modulates CFTR Biogenesis: Is the Pathway Druggable?
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DOI：
10.2174/1389450116666150531152236
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发表时间：
2015-01-01
影响因子：
3.2
通讯作者：
Frizzell, Raymond A.
中科院分区：
文献类型：
--
作者：
Ahner, Annette;Frizzell, Raymond A.
SUMO化途径参与调节许多不同的细胞功能，包括核内和核外功能。因此，SUMO通路组分与囊性纤维化、癌症和神经退行性疾病等多种疾病有关并不奇怪。因此，SUMO化途径的组分应该为操作提供有效的治疗靶点。虽然相关的泛素化系统包括大量的酶作为潜在的药物靶点，但只有少数组分构成SUMO化级联。尽管这加剧了靶标冗余的问题，但它可能使实现药物特异性的潜力复杂化。针对SUMO通路组分的小分子抑制剂的开发处于早期阶段。本文综述了该途径的概况，并总结了针对单个SUMO化途径组分的药物开发工作，重点是CFTR蛋白加工可能受到的影响。
The SUMOylation pathway is involved in the regulation of numerous and diverse cellular functions, nuclear as well as extra-nuclear. Thus, it is not surprising that SUMO pathway components are implicated in diseases as diverse as cystic fibrosis, cancer and neurodegenerative diseases. Therefore, the components of the SUMOylation pathway should provide valid therapeutic targets for manipulation. While the related ubiquitylation system encompasses a vast number of enzymes as potential drug targets, there are only a handful of components that comprise the SUMOylation cascade. Whereas this alleviates the problem of target redundancy, it may complicate the potential to achieve drug specificity. The development of small molecule inhibitors aimed at SUMO pathway components is in its early stages. This review provides an outline of the pathway and summarizes drug development efforts targeted at individual SUMOylation pathway components, with an emphasis on how CFTR protein processing may be affected.