School of Life Science and Technology, Henan Mental Hospital, Xinxiang Medical University, Number 601, Jinsui Road, Xinxiang, 453003, Henan, China.
Henan Key Laboratory of Medical Tissue Regeneration, Xinxiang, 453003, Henan, China.
Mol Neurobiol. 2022 Feb;59(2):968-982. doi: 10.1007/s12035-021-02638-w. Epub 2021 Nov 23.
Neurological disorders are primarily diseases with sophisticated etiology that are always refractory and recrudescent. The major obstruction to effective therapies for neurological disorders is the poor understanding of their pathogenic mechanisms. CRISPR-Cas9 technology, which allows precise and effective gene editing in almost any cell type and organism, is accelerating the pace of basic biological research. An increasing number of groups are focusing on uncovering the molecular mechanisms of neurological disorders and developing novel therapies using the CRISPR-Cas9 system. This review highlights the application of CRISPR-Cas9 technology in the treatment of neurological disorders, including Alzheimer's disease, amyotrophic lateral sclerosis and/or frontotemporal dementia, Duchenne muscular dystrophy, Dravet syndrome, epilepsy, Huntington's disease, and Parkinson's disease. Hopefully, it will improve our understanding of neurological disorders and give insights into future treatments for neurological disorders.
神经紊乱疾病主要是病因复杂、难治且易复发的疾病。神经紊乱疾病有效治疗的主要障碍是对其发病机制的了解不足。CRISPR-Cas9 技术几乎可以在任何细胞类型和生物体中进行精确有效的基因编辑，它正在加速基础生物学研究的步伐。越来越多的研究小组开始利用 CRISPR-Cas9 系统来揭示神经紊乱疾病的分子机制并开发新的治疗方法。本综述重点介绍了 CRISPR-Cas9 技术在神经紊乱疾病治疗中的应用，包括阿尔茨海默病、肌萎缩侧索硬化症和/或额颞叶痴呆、杜氏肌营养不良症、德拉韦特综合征、癫痫、亨廷顿病和帕金森病。希望这能增进我们对神经紊乱疾病的了解，并为神经紊乱疾病的未来治疗提供思路。
