Chacko Leena, Chaudhary Anupama, Singh Birbal, Dewanjee Saikat, Kandimalla Ramesh
BioAnalytical Lab, Meso Scale Discovery, 1601 Research Blvd, Rockville, MD, USA.
Orinin-BioSystems, LE-52, Lotus Road 4, CHD City, Karnal, Haryana 132 001, India.
Drug Discov Today. 2023 Aug;28(8):103652. doi: 10.1016/j.drudis.2023.103652. Epub 2023 Jun 7.
Alzheimer's disease (AD) is a progressive neurodegenerative disorder with no known cure, which has prompted the exploration of novel therapeutic approaches. The clustered regularly interspaced palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) tool has generated significant interest for its potential in AD therapeutics by correcting faulty genes. Our report comprehensively reviews emerging applications for CRISPR-Cas9 in developing in vitro and in vivo models for AD research and therapeutics. We further assess its ability to identify and validate genetic markers and potential therapeutic targets for AD. Moreover, we review the current challenges and delivery strategies for the in vivo application of CRISPR-Cas9 in AD therapeutics.
阿尔茨海默病（AD）是一种无法治愈的进行性神经退行性疾病，这促使人们探索新的治疗方法。成簇规律间隔短回文重复序列（CRISPR）-CRISPR相关蛋白9（Cas9）工具因其通过纠正缺陷基因在AD治疗中的潜力而引起了极大关注。我们的报告全面回顾了CRISPR-Cas9在开发用于AD研究和治疗的体外和体内模型中的新兴应用。我们进一步评估了其识别和验证AD遗传标记及潜在治疗靶点的能力。此外，我们还回顾了CRISPR-Cas9在AD治疗体内应用的当前挑战和递送策略。
