Zaib Sumera, Saleem Mushtaq A, Khan Imtiaz
Department of Biochemistry, Faculty of Life Sciences, University of Central Punjab, Lahore-54590, Pakistan.
Ghazi National Institute of Engineering & Sciences -GNIES, Dera Ghazi Khan-32200, Pakistan.
Mini Rev Med Chem. 2022;22(3):410-421. doi: 10.2174/1389557521666210913112030.
The ability to engineer biological systems and organisms holds enormous potential for applications across basic science, medicine, and biotechnology. Over the past few decades, the development of CRISPR (clustered regularly interspaced short palindromic repeat) has revolutionized the whole genetic engineering process utilizing the principles of Watson-Crick base pairing. CRISPRCas9 technology offers the simplest, fastest, most versatile, reliable, and precise method of genetic manipulation, thus enabling geneticists and medical researchers to edit parts of the genome by removing, adding, or altering sections of the DNA sequence. The current review focuses on the applications of CRISPR-Cas9 in the field of medical research. Compared with other gene-editing technologies, CRISPR/Cas9 demonstrates numerous advantages for the treatment of various medical conditions, including cancer, hepatitis B, cardiovascular diseases, or even high cholesterol. Given its promising performance, CRISPR/Cas9 gene-editing technology will surely help in the therapy of several disorders while addressing the issues pertaining to the minimization of the off-target effects of gene editing and incomplete matches between sgRNA and genomic DNA by Cas9.
设计生物系统和生物体的能力在基础科学、医学和生物技术等领域的应用中具有巨大潜力。在过去几十年里，CRISPR（成簇规律间隔短回文重复序列）的发展利用沃森-克里克碱基配对原则彻底改变了整个基因工程过程。CRISPRCas9技术提供了最简单、最快、最通用、可靠且精确的基因操作方法，从而使遗传学家和医学研究人员能够通过去除、添加或改变DNA序列片段来编辑基因组的部分内容。本综述聚焦于CRISPR-Cas9在医学研究领域的应用。与其他基因编辑技术相比，CRISPR/Cas9在治疗各种疾病方面展现出诸多优势，包括癌症、乙型肝炎、心血管疾病，甚至高胆固醇。鉴于其出色的表现，CRISPR/Cas9基因编辑技术必将有助于治疗多种疾病，同时解决与基因编辑脱靶效应最小化以及sgRNA与基因组DNA之间不完全匹配相关的问题。
