拓展CRISPR/Cas技术的视野：临床进展、治疗应用及基因治疗中的挑战
Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy.
作者信息
Bairqdar Ahmad, Karitskaya Polina E, Stepanov Grigory A
机构信息
Institute of Chemical Biology and Fundamental Medicine of the Siberian Branch of the Russian Academy of Sciences, Novosibirsk 630090, Russia.
Department of Natural Sciences, Novosibirsk State University, Novosibirsk 630090, Russia.
出版信息
Int J Mol Sci. 2024 Dec 12;25(24):13321. doi: 10.3390/ijms252413321.
CRISPR-Cas technology has transformed the field of gene editing, opening new possibilities for treatment of various genetic disorders. Recent years have seen a surge in clinical trials using CRISPR-Cas-based therapies. This review examines the current landscape of CRISPR-Cas implementation in clinical trials, with data from key registries, including the Australian New Zealand Clinical Trials Registry, the Chinese Clinical Trial Register, and ClinicalTrials.gov. Emphasis is placed on the mechanism of action of tested therapies, the delivery method, and the most recent findings of each clinical trial.
CRISPR-Cas技术已经改变了基因编辑领域，为治疗各种遗传疾病开辟了新的可能性。近年来，使用基于CRISPR-Cas的疗法的临床试验激增。本综述利用来自主要注册机构的数据，包括澳大利亚新西兰临床试验注册中心、中国临床试验注册中心和美国国立医学图书馆临床试验数据库，审视了CRISPR-Cas在临床试验中的应用现状。重点关注受试疗法的作用机制、给药方法以及每项临床试验的最新发现。
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