Department of Oncology, Affiliated Hospital of Xuzhou Medical University, Xuzhou 221002, China.
Department of Dermatology, The Affiliated Huai'an Hospital of Xuzhou Medical University, The Second People's Hospital of Huai'an, No. 62, Huaihai Road(S.), Huai'an 223002, China.
Anticancer Agents Med Chem. 2019;19(16):1912-1919. doi: 10.2174/1871520619666191014103711.
Genome editing refers to changing the genome sequence of an organism by knockout, insertion, and site mutation, resulting in changes in the genetic information of the organism. The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein-9 nuclease (Cas9) system is a genome editing technique developed by the acquired immune system in the microbes, such as bacteria and archaebacteria, which targets and edits genome sequences according to the principle of complementary base pairing. This technique can be used to edit endogenous genomic DNA sequences in organisms accurately and has been widely used in fields, such as biotechnology, cancer gene therapy, and dermatology. In this review, we summarize the history, structure, mechanism, and application of CRISPR/Cas9 in gene therapy and dermatological diseases.
基因组编辑是指通过敲除、插入和位点突变来改变生物体的基因组序列，从而导致生物体遗传信息的改变。成簇规律间隔短回文重复序列（CRISPR）/ CRISPR 相关蛋白 9 核酸酶（Cas9）系统是一种由细菌和古细菌等微生物获得性免疫系统开发的基因组编辑技术，它根据互补碱基配对的原理靶向和编辑基因组序列。该技术可用于精确编辑生物体的内源性基因组 DNA 序列，已广泛应用于生物技术、癌症基因治疗和皮肤病等领域。本文综述了 CRISPR/Cas9 在基因治疗和皮肤病中的历史、结构、机制和应用。
