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近年来,可编程核酸酶介导的基因编辑技术迅猛发展。CRISPR/Cas9技术源于细菌和古生菌的适应性免疫系统,主要由Cas9内切酶和向导RNA(guide RNA,gRNA)组成。Cas9内切酶在gRNA的指导下造成DNA的双链断裂,从而使研究人员能够精准高效地操纵特定基因组位点。同时,该系统可以揭示基因在疾病进程中所扮演的未知角色,在临床治疗中有应用潜能。现总结了CRISPR/Cas9技术在疾病模型构建与基因修复领域应用的研究进展。
In recent years, programmable nuclease-mediated gene editing technology is developing rapidly. CRISPR / Cas9 technology is derived from the adaptive immune system of bacteria and archaea, and consists mainly of Cas9 endonuclease and guide RNA (gRNA). Cas9 endonucleases cause DNA double-strand breaks under the guidance of gRNAs, enabling researchers to manipulate specific genomic sites precisely and efficiently. At the same time, the system can reveal the unknown role played by genes in the disease process and has potential application in clinical treatment. Now summarizes the CRISPR / Cas9 technology in disease model construction and application of gene repair research progress.