CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR associated Protein 9), basically a bacterial immune system is now widely applicable to engineer genomes of a number of cells and organisms because of its simplicity and robustness. In research avenue the system has been optimized to regulate gene expression, modify epigenome and edit target locus. These applications make CRISPR/Cas9, a technology of choice to edit disease causing mutations as well as the epigenome more efficiently than ever before. Meanwhile its application in in vivo and ex vivo cells is encouraging the scientific community for more vigorous gene therapy and in clinical setups for therapeutic genome editing. Here we review the recent advances that CRISPR-Cas9 mediated genome editing has achieved and is reported in previous studies and address the challenges associated with it.

中文翻译：

---

使用最先进的技术治疗遗传疾病。

CRISPR-Cas9（成簇的定期间隔短回文重复序列-CRISPR 相关蛋白 9），基本上是一种细菌免疫系统，由于其简单性和稳健性，现在广泛适用于许多细胞和生物体的基因组工程。在研究方面，该系统已被优化以调节基因表达、修改表观基因组和编辑目标基因座。这些应用使 CRISPR/Cas9 成为一种编辑导致疾病的突变以及表观基因组的首选技术，比以往任何时候都更有效。同时，它在体内和离体细胞中的应用正在鼓励科学界进行更有力的基因治疗和治疗性基因组编辑的临床设置。