Cheng Lin, Liu Ziying, Shen Chunxiao, Xiong Yinyi, Shin Sang Yol, Hwang Yong, Yang Seung-Bum, Chen Zhiying, Zhang Xiaorong
Department of Neurology, Affiliated Hospital of Jiujiang University, Jiujiang, Jiangxi, China.
Jiujiang Clinical Precision Medicine Research Center, Jiujiang, Jiangxi, China.
CNS Neurosci Ther. 2025 Jan;31(1):e70208. doi: 10.1111/cns.70208.
Adenosine deaminase action on RNA 1 (ADAR1) can convert the adenosine in double-stranded RNA (dsRNA) molecules into inosine in a process known as A-to-I RNA editing. ADAR1 regulates gene expression output by interacting with RNA and other proteins; plays important roles in development, including growth; and is linked to innate immunity, tumors, and central nervous system (CNS) diseases.
In recent years, the role of ADAR1 in tumors has been widely discussed, but its role in CNS diseases has not been reviewed. It is worth noting that recent studies have shown ADAR1 has great potential in the treatment of neurodegenerative diseases, but the mechanisms are still unclear. Therefore, it is necessary to elaborate on the role of ADAR1 in CNS diseases.
Here, we focus on the effects and mechanisms of ADAR1 on CNS diseases such as Aicardi-AicardiGoutières syndrome, Alzheimer's disease, Parkinson's disease, glioblastoma, epilepsy, amyotrophic lateral sclerosis, and autism. We also evaluate the impact of ADAR1-based treatment strategies on these diseases, with a particular focus on the development and treatment strategies of new technologies such as microRNAs, nanotechnology, gene editing, and stem cell therapy. We hope to provide new directions and insights for the future development of ADAR1 gene editing technology in brain science and the treatment of CNS diseases.
RNA 特异性腺苷脱氨酶 1(ADAR1)可在一个称为 A 到 I RNA 编辑的过程中将双链 RNA(dsRNA)分子中的腺苷转化为肌苷。ADAR1 通过与 RNA 和其他蛋白质相互作用来调节基因表达输出;在包括生长在内的发育过程中发挥重要作用;并与先天免疫、肿瘤和中枢神经系统(CNS)疾病相关。
近年来,ADAR1 在肿瘤中的作用已被广泛讨论,但其在中枢神经系统疾病中的作用尚未得到综述。值得注意的是,最近的研究表明 ADAR1 在神经退行性疾病的治疗中具有巨大潜力,但其机制仍不清楚。因此,有必要详细阐述 ADAR1 在中枢神经系统疾病中的作用。
在此,我们重点关注 ADAR1 对诸如 Aicardi - AicardiGoutières 综合征、阿尔茨海默病、帕金森病、胶质母细胞瘤、癫痫、肌萎缩侧索硬化症和自闭症等中枢神经系统疾病的影响及机制。我们还评估了基于 ADAR1 的治疗策略对这些疾病的影响,特别关注 microRNAs、纳米技术、基因编辑和干细胞治疗等新技术的开发和治疗策略。我们希望为 ADAR1 基因编辑技术在脑科学和中枢神经系统疾病治疗中的未来发展提供新的方向和见解。