CRISPR/Cas9 技术作为一种强大的基因治疗分子工具。
CRISPR/Cas9 technology as a potent molecular tool for gene therapy.
机构信息
Cellular and Molecular Biology Research Center, Health Research Institute, Babol University of Medical Sciences, Babol, Iran.
Cancer & Immunology Research Center, Kurdistan University of Medical Sciences, Sanandaj, Iran.
出版信息
J Cell Physiol. 2019 Aug;234(8):12267-12277. doi: 10.1002/jcp.27972. Epub 2019 Jan 30.
Clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9 (CRISPR-Cas9) is an RNA-guided gene editing tool which offers several advantageous characteristics in comparison with the conventional methods (e.g., zinc finger nucleases and transcription activator-like effector nucleases) such as cost-effectiveness, flexibility, and being easy-to-use. Despite some limitations such as efficient delivery and safety, CRISPR-Cas9 is still the most convenient tool for gene editing purposes. Due to the potential capability of the CRISPR-Cas9 system in genome editing and correction of casual mutations, it can be considered as a possible therapeutic system in the treatment of disorders associated with the genome mutations and in particular cancer treatment. In this review, we will discuss CRISPR-Cas-based gene editing along with its classifications and mechanism of action. Furthermore, the therapeutic application of the CRISPR-Cas9 system in mutational disorders, delivery systems, as well as its advantages and limitations with a special emphasis on cancer treatment will be discussed.
成簇规律间隔短回文重复/CRISPR 相关核酸酶 9(CRISPR-Cas9)是一种 RNA 指导的基因编辑工具,与传统方法(例如锌指核酸酶和转录激活因子样效应核酸酶)相比具有几个优势特征,如成本效益、灵活性和易于使用。尽管存在一些局限性,如有效递送和安全性,CRISPR-Cas9 仍然是基因编辑目的最方便的工具。由于 CRISPR-Cas9 系统在基因组编辑和纠正偶然突变方面的潜在能力,它可以被视为治疗与基因组突变相关的疾病的一种可能的治疗系统,特别是癌症治疗。在这篇综述中,我们将讨论基于 CRISPR 的基因编辑及其分类和作用机制。此外,还将讨论 CRISPR-Cas9 系统在突变性疾病、递送系统中的治疗应用,以及其优点和局限性,特别强调癌症治疗。