摘要
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.
基金
This work was supported by the National Institutes of Health(NIH)(Grant No.AI138203-3)
the American Association of Immunologists through a Careers in Immunology Fellowship.This work was also supported by the National Natural Science Foundation of China(Grants No.82020108021 and 32000033).
