期刊
PRECISION CLINICAL MEDICINE
卷 4, 期 3, 页码 179-191出版社
OXFORD UNIV PRESS
DOI: 10.1093/pcmedi/pbab014
关键词
gene editing; clinical trials; hereditary diseases; cancer; gene therapy; viral vectors
资金
- National Institutes of Health (NIH) [AI138203-3]
- American Association of Immunologists through a Careers in Immunology Fellowship
- National Natural Science Foundation of China [82020108021, 32000033]
CRISPR-Cas systems are efficient tools for gene editing in various applications, yet challenges such as editing efficiency and delivery difficulties still persist. Solutions are needed to address these obstacles in order to optimize their use in clinical treatments and precision medicine.
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.
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