National Engineering Laboratory for Deep Process of Rice and Byproducts, College of Food Science and Engineering, Central South University of Forestry and Technology, Changsha 410004, Hunan, China.
Institute of Oncology, Affiliated Tumor Hospital of Nantong University, Nantong 226631, China.
J Adv Res. 2022 Sep;40:135-152. doi: 10.1016/j.jare.2021.11.018. Epub 2021 Dec 4.
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is derived from the bacterial innate immune system and engineered as a robust gene-editing tool. Due to the higher specificity and efficiency of CRISPR/Cas9, it has been widely applied to many genetic and non-genetic disease, including cancers, genetic hemolytic diseases, acquired immunodeficiency syndrome, cardiovascular diseases, ocular diseases, and neurodegenerative diseases, and some X-linked diseases. Furthermore, in terms of the therapeutic strategy of cancers, many researchers used the CRISPR/Cas9 technique to cure or alleviate cancers through different approaches, such as gene therapy and immune therapy.
Here, we conclude the recent application and clinical trials of CRISPR/Cas9 in non-cancerous diseases and cancers and pointed out some of the problems to be solved.
CRISPR/Cas9, derived from the microbial innate immune system, is developed as a robust gene-editing tool and has been applied widely. Due to its high accuracy and efficiency, CRISPR/Cas9 techniques may provide a great chance to treat some gene-related diseases by disrupting, inserting, correcting, replacing, or blocking genes for clinical application with gene therapy.
CRISPR（Clustered Regularly Interspaced Short Palindromic Repeats）来源于细菌的先天免疫系统，被设计为一种强大的基因编辑工具。由于 CRISPR/Cas9 的更高特异性和效率，它已被广泛应用于许多遗传和非遗传疾病，包括癌症、遗传性溶血性疾病、获得性免疫缺陷综合征、心血管疾病、眼部疾病和神经退行性疾病，以及一些 X 连锁疾病。此外，在癌症的治疗策略方面，许多研究人员通过不同的方法，如基因治疗和免疫治疗，使用 CRISPR/Cas9 技术来治疗或缓解癌症。
本文总结了 CRISPR/Cas9 在非癌症疾病和癌症中的最新应用和临床试验，并指出了一些需要解决的问题。
CRISPR/Cas9 来源于微生物的先天免疫系统，被开发为一种强大的基因编辑工具，并已得到广泛应用。由于其高精度和高效率，CRISPR/Cas9 技术可能通过基因治疗来干扰、插入、纠正、替换或阻断基因，为一些基因相关疾病的临床应用提供了很好的机会。
