CRISPR/Cas9介导的疾病模型构建与基因修复研究进展

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。

The Progress of CRISPR/Cas9 for Disease Modeling and Gene Correction

Recently, genomic engineering technology using programmable nucleases boomed. The CRISPR/Cas9 system that consists of endonuclease Cas9 and guide RNA (gRNA) is derived from the adaptive bacterial and archaea immune system. Cas9 endonuclease introduces the DNA double-stranded break with the guidance of gRNA, therefore it enables researchers to precisely and efficiently manipulate specific genomic locus. Meanwhile,this system can reveal the unknown roles of genes in disease processes, and has great potential for clinical therapeutic applications. Here current progresses in disease modeling and gene correction by CRISPR/Cas9 were reviewed.