Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics.
Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics.
复制标题
DOI：
10.1093/pcmedi/pbab014
复制
发表时间：
2021-09
影响因子：
5.3
通讯作者：
Lin P
中科院分区：
文献类型：
--
作者：
Liu W;Li L;Jiang J;Wu M;Lin P
规则间隔短回文重复序列（CRISPR）相关系统（Cas）是针对实验室研究，农业工程，生物技术和人类疾病治疗的特定基因的有效工具。Cas9是迄今为止使用最广泛的基因编辑核酸酶，在治疗遗传性疾病、病毒感染、癌症等方面显示出巨大的前景。最近的报告显示，其他类型的CRISPR-Cas系统也可能具有惊人的潜力，可以作为各种应用的基因编辑工具加入竞争。尽管在基础研究和临床试验方面取得了快速进展，但一些潜在的问题仍然存在持续的重大挑战，例如编辑效率，递送相对困难，脱靶效应，免疫原性，本文总结了CRISPR-Cas从实验室到床旁的应用，并强调了目前可能限制CRISPR-Cas系统作为基因工程的应用的障碍。编辑精准医学的工具包，并提供一些可能有助于应对这些挑战和促进技术发展的观点。CRISPR-Cas系统作为一种强大的基因编辑方法，将为许多目前患有不治之症的个体的临床治疗带来巨大希望。
Clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems (Cas) are efficient tools for targeting specific genes for laboratory research, agricultural engineering, biotechnology, and human disease treatment. Cas9, by far the most extensively used gene-editing nuclease, has shown great promise for the treatment of hereditary diseases, viral infection, cancers, and so on. Recent reports have revealed that some other types of CRISPR-Cas systems may also have surprising potential to join the fray as gene-editing tools for various applications. Despite the rapid progress in basic research and clinical tests, some underlying problems present continuous, significant challenges, such as editing efficiency, relative difficulty in delivery, off-target effects, immunogenicity, etc. This article summarizes the applications of CRISPR-Cas from bench to bedside and highlights the current obstacles that may limit the usage of CRISPR-Cas systems as gene-editing toolkits in precision medicine and offer some viewpoints that may help to tackle these challenges and facilitate technical development. CRISPR-Cas systems, as a powerful gene-editing approach, will offer great hopes in clinical treatments for many individuals with currently incurable diseases.