作用于 RNA 1 (ADAR1) 的腺苷脱氨酶将双链 RNA (dsRNA) 分子中的腺苷转化为肌苷，这一过程称为 A 到 I 编辑。人类和小鼠的 ADAR1 缺陷会导致严重的炎症性疾病，其特征是自发诱导先天免疫。在缺乏 ADAR1 的细胞中，未经编辑的 RNA 会激活 RNA 传感器。其中包括诱导细胞因子表达的黑色素瘤分化相关基因 5 (MDA5)，特别是 I 型干扰素 (IFN)、蛋白激酶 R (PKR)、寡腺苷酸合成酶 (OAS) 和 Z-DNA/RNA 结合蛋白 1 (ZBP1) ）。被 ADAR1“解散”的免疫原性 RNA 可能包括来自重复元件和其他长双链体 RNA 的转录物。在这里，我们回顾这些最近的基本发现并讨论对人类疾病的影响。一些肿瘤依赖 ADAR1 来逃避免疫监视，从而开启了使用 ADAR1 抑制剂进行抗癌治疗的可能性。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。

Adenosine deaminase acting on RNA 1 (ADAR1) converts adenosine to inosine in double-stranded RNA (dsRNA) molecules, a process known as A-to-I editing. ADAR1 deficiency in humans and mice results in profound inflammatory diseases characterised by the spontaneous induction of innate immunity. In cells lacking ADAR1, unedited RNAs activate RNA sensors. These include melanoma differentiation-associated gene 5 (MDA5) that induces the expression of cytokines, particularly type I interferons (IFNs), protein kinase R (PKR), oligoadenylate synthase (OAS), and Z-DNA/RNA binding protein 1 (ZBP1). Immunogenic RNAs 'defused' by ADAR1 may include transcripts from repetitive elements and other long duplex RNAs. Here, we review these recent fundamental discoveries and discuss implications for human diseases. Some tumours depend on ADAR1 to escape immune surveillance, opening the possibility of unleashing anticancer therapies with ADAR1 inhibitors.Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.