CRISPR/Cas9系统在临床医学研究的应用与改进

Application and Improvement of the CRISPR/Cas9 System in Clinical Medical Research

癌症与遗传疾病等难治性疾病的发生发展一般是多因素协同的结果,涉及复杂的信号网络及多生物分子的相互作用。了解其中驱动的关键元素有助于为临床上的治疗和研究打开新的突破口。然而,探究驱动元素用于难治性疾病治疗的挑战之一是缺乏方便、可编程的基因编辑工具。近年来,新型的CRISPR/Cas9系统由于其组分简单、基因编辑效率高等特点逐渐成为临床医学研究中应用最为广泛的基因编辑工具。本文介绍了CRISPR系统应用于临床研究中的相应进展和潜在挑战。阐述了基于CRISPR系统sgRNA序列重构能改变靶向性及系统本身的可编程性等特性,其可在进行适当的改造和修饰后实现对活细胞染色体的实时成像,用以了解生物体在面临外界刺激时基因组的时空调节。评估了CRISPR系统在基因筛选、免疫治疗和遗传疾病治疗方面的重大价值,尤其是CRISPR系统进行相应改造后系统用在临床研究中安全性与功能性的提升。介绍了CRISPR系统在临床研究中的应用障碍、局限以及对其相应的优化改造,展望CRISPR/Cas9基因编辑技术的应用前景及其在临床医学领域的发展优势,以期能为CRISPR系统的进一步应用与优化提供参考。

The occurrence and development of refractory diseases,such as cancer and genetic diseases,is generally the result of multi-factor coordination,involving complex signal networks and multi-biomolecular interactions.Understanding the key driving elements is helpful to open a new breakthrough for clinical treatment and research.However,one of the challenges in exploring drivers for refractory diseases is the lack of convenient,programmable gene-editing tools.In recent years,the new CRISPR/Cas9 system has gradually become the most widely used gene editing tool in clinical medical research due to its simple composition and high gene editing efficiency.This paper introduces the progress and potential challenges of the CRISPR system in clinical research.The CRISPR system can realize real-time imaging of living cell chromosomes after appropriate modification,so as to understand the temporal and spatial regulation of the genome in the face of external stimuli.The important value of the CRISPR system in gene screening,immunotherapy and the treatment of genetic diseases was evaluated,especially the improvement of safety and function of the CRISPR system in clinical research after corresponding modification.In this paper,the obstacles and limitations of the CRISPR system application in clinical research as well as the corresponding optimization of the CRISPR system were introduced,and the application prospect of the CRISPR/Cas9 gene editing technology and its development advantages in the field of clinical medicine were prospected,in order to provide a reference for the further application and optimization of the CRISPR system.