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NEMO 将核因子-κB 与人类疾病联系起来。

NEMO Links Nuclear Factor-κB to Human Diseases.

机构信息

Institute of Experimental Internal Medicine, Otto von Guericke University, Magdeburg, Germany.

Institute of Experimental Internal Medicine, Otto von Guericke University, Magdeburg, Germany.

出版信息

Trends Mol Med. 2017 Dec;23(12):1138-1155. doi: 10.1016/j.molmed.2017.10.004. Epub 2017 Nov 8.

Abstract

The nuclear factor (NF)-κB essential modulator (NEMO) is a key regulator in NF-κB-mediated signaling. By transmitting extracellular or intracellular signals, NEMO can control NF-κB-regulated genes. NEMO dysfunction is associated with inherited diseases such as incontinentia pigmenti (IP), ectodermal dysplasia, anhidrotic, with immunodeficiency (EDA-ID), and some cancers. We focus on molecular studies, human case reports, and mouse models emphasizing the significance of NEMO molecular interactions and modifications in health and diseases. This knowledge opens new opportunities to engineer suitable drugs that may putatively target precise NEMO functions attributable to various diseases, while leaving other functions intact, and eliminating cytotoxicity. Indeed, with the advent of novel gene editing tools such as clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas)9, treating some inherited diseases may in the long run, become a reality.

摘要

核因子 (NF)-κB 必需调节剂 (NEMO) 是 NF-κB 介导的信号转导中的关键调节剂。通过传递细胞外或细胞内信号,NEMO 可以控制 NF-κB 调节的基因。NEMO 功能障碍与遗传性疾病有关,如色素失禁症 (IP)、外胚层发育不良、无汗性、免疫缺陷 (EDA-ID) 和一些癌症。我们专注于分子研究、人类病例报告和小鼠模型,强调 NEMO 分子相互作用和修饰在健康和疾病中的重要性。这一知识为设计合适的药物提供了新的机会,这些药物可能针对各种疾病的特定 NEMO 功能,而不影响其他功能,并消除细胞毒性。事实上,随着新型基因编辑工具如成簇规律间隔短回文重复 (CRISPR)/CRISPR 相关蛋白 (Cas)9 的出现,从长远来看,治疗某些遗传性疾病可能成为现实。

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