Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) is one of the most versatile and efficient gene editing technologies, which is derived from adaptive immune strategies for bacteria and archaea. With the remarkable development of programmable nuclease-based genome engineering these years, CRISPR-Cas9 system has developed quickly in recent 5 years and has been widely applied in countless areas, including genome editing, gene function investigation and gene therapy both in vitro and in vivo. In this paper, we briefly introduce the mechanisms of CRISPR-Cas9 tool in genome editing. More importantly, we review the recent therapeutic application of CRISPR-Cas9 in various diseases, including hematologic diseases, infectious diseases and malignant tumor. Finally, we discuss the current challenges and consider thoughtfully what advances are required in order to further develop the therapeutic application of CRISPR-Cas9 in the future.

中文翻译：

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CRISPR-Cas9的治疗应用进展。

簇状规则间隔的短回文重复序列（CRISPR）相关蛋白9（Cas9）是最通用，最有效的基因编辑技术之一，它源自细菌和古细菌的适应性免疫策略。近年来，随着基于可编程核酸酶的基因组工程的显着发展，CRISPR-Cas9系统在近5年中发展迅速，已广泛应用于无数领域，包括基因组编辑，基因功能研究和体内外基因治疗。在本文中，我们简要介绍了CRISPR-Cas9工具在基因组编辑中的机制。更重要的是，我们综述了CRISPR-Cas9在各种疾病（包括血液系统疾病，传染病和恶性肿瘤）中的最新治疗应用。最后，