Molecular Systematics Laboratory, Parasitology Department, Pasteur Institute of Iran, Tehran, Iran.
Molecular Medicine Department, Biotechnology Research Center (BRC), Pasteur Institute of Iran, Tehran, Iran.
Iran Biomed J. 2023 Sep 1;27(5):219-46. doi: 10.61186/ibj.27.5.219. Epub 2023 Jun 18.
Programmable nucleases are powerful genomic tools for precise genome editing. These tools precisely recognize, remove, or change DNA at a defined site, thereby, stimulating cellular DNA repair pathways that can cause mutations or accurate replacement or deletion/insertion of a sequence. CRISPR-Cas9 system is the most potent and useful genome editing technique adapted from the defense immune system of certain bacteria and archaea against viruses and phages. In the past decade, this technology made notable progress, and at present, it has largely been used in genome manipulation to make precise gene editing in plants, animals, and human cells. In this review, we aim to explain the basic principle, mechanisms of action, and applications of this system in different areas of medicine, with emphasizing on the detection and treatment of parasitic diseases.
可编程核酸酶是精确基因组编辑的强大基因组工具。这些工具可以精确地识别、移除或改变特定位置的 DNA,从而刺激细胞的 DNA 修复途径,导致突变或准确的替换或序列的缺失/插入。CRISPR-Cas9 系统是最有效和有用的基因组编辑技术,它源自某些细菌和古细菌的防御免疫系统,以抵御病毒和噬菌体。在过去的十年中,这项技术取得了显著的进展,目前已广泛用于基因组操作,以实现植物、动物和人类细胞的精确基因编辑。在这篇综述中,我们旨在解释该系统在医学不同领域的基本原理、作用机制和应用,重点介绍寄生虫病的检测和治疗。