Spinal Cord Injury Center, Heidelberg University Hospital, Heidelberg, Germany.
Second Clinical Medical College of Guangzhou University of Chinese Medicine, Guangdong Provincial Hospital of Chinese Medicine,Guangzhou, China.
Brief Funct Genomics. 2020 May 20;19(3):209-214. doi: 10.1093/bfgp/elaa001.
Clustered regularly interspaced short palindromic repeats (CRISPR) is described as RNA mediated adaptive immune system defense, which is naturally found in bacteria and archaea. CRISPR-Cas9 has shown great promise for cancer treatment in cancer immunotherapy, manipulation of cancer genome and epigenome and elimination or inactivation of carcinogenic viral infections. However, many challenges remain to be addressed to increase its efficacy, including off-target effects, editing efficiency, fitness of edited cells, immune response and delivery methods. Here, we explain CRISPR-Cas classification and its general function mechanism for gene editing. Then, we summarize these preclinical CRISPR-Cas9-based therapeutic strategies against cancer. Moreover, the challenges and improvements of CRISPR-Cas9 clinical applications will be discussed.
成簇规律间隔短回文重复序列 (CRISPR) 被描述为 RNA 介导的适应性免疫系统防御,它自然存在于细菌和古菌中。CRISPR-Cas9 在癌症免疫治疗、癌症基因组和表观基因组的操作以及致癌病毒感染的消除或失活方面显示出巨大的应用潜力。然而,为了提高其疗效,仍有许多挑战需要解决,包括脱靶效应、编辑效率、编辑细胞的适应性、免疫反应和递送方法。在这里,我们解释了 CRISPR-Cas 的分类及其用于基因编辑的一般功能机制。然后,我们总结了这些基于 CRISPR-Cas9 的治疗癌症的临床前策略。此外,还将讨论 CRISPR-Cas9 临床应用的挑战和改进。