Non-viral and viral delivery systems for CRISPR-Cas9 technology in the biomedical field

The clustered regularly interspaced short palindromic repeats（CRISPR）-associated protein 9（CRISPR-Cas9） system provides a novel genome editing technology that can precisely target a genomic site to disrupt or repair a specific gene. Some CRISPR-Cas9 systems from different bacteria or artificial vari...

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Bibliographic Details
Published inScience China. Life sciences Vol. 60; no. 5; pp. 458 - 467
Main Authors He, Zhi-Yao, Men, Ke, Qin, Zhou, Yang, Yang, Xu, Ting, Wei, Yu-Quan
Format Journal Article
LanguageEnglish
Published Beijing Science China Press 01.05.2017
Springer Nature B.V
Subjects
Animal models
Animals
Biomedical and Life Sciences
Biomedical Research - methods
CRISPR
CRISPR-Cas Systems
Editing
Endonucleases - genetics
Endonucleases - metabolism
Gene Editing - methods
Gene therapy
Gene Transfer Techniques
Genes
Genome editing
Genomes
Homology
Humans
Life Sciences
Models, Genetic
mRNA
Nuclease
Review
Reviews
RNA, Guide, CRISPR-Cas Systems - genetics
RNA, Guide, CRISPR-Cas Systems - metabolism
Therapeutic targets
Transcription
Viruses - genetics
传递系统
医学领域
基因治疗
基因组
生物学家
相关蛋白
编辑技术
非病毒载体
genome editing
CRISPR
gene therapy
Cas9
viral vector
non-viral vector
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Summary:The clustered regularly interspaced short palindromic repeats（CRISPR）-associated protein 9（CRISPR-Cas9） system provides a novel genome editing technology that can precisely target a genomic site to disrupt or repair a specific gene. Some CRISPR-Cas9 systems from different bacteria or artificial variants have been discovered or constructed by biologists, and Cas9 nucleases and single guide RNAs（sgRNA） are the major components of the CRISPR-Cas9 system. These Cas9 systems have been extensively applied for identifying therapeutic targets, identifying gene functions, generating animal models, and developing gene therapies.Moreover, CRISPR-Cas9 systems have been used to partially or completely alleviate disease symptoms by mutating or correcting related genes. However, the efficient transfer of CRISPR-Cas9 system into cells and target organs remains a challenge that affects the robust and precise genome editing activity. The current review focuses on delivery systems for Cas9 mRNA, Cas9 protein, or vectors encoding the Cas9 gene and corresponding sgRNA. Non-viral delivery of Cas9 appears to help Cas9 maintain its on-target effect and reduce off-target effects, and viral vectors for sgRNA and donor template can improve the efficacy of genome editing and homology-directed repair. Safe, efficient, and producible delivery systems will promote the application of CRISPR-Cas9 technology in human gene therapy.
Bibliography:The clustered regularly interspaced short palindromic repeats（CRISPR）-associated protein 9（CRISPR-Cas9） system provides a novel genome editing technology that can precisely target a genomic site to disrupt or repair a specific gene. Some CRISPR-Cas9 systems from different bacteria or artificial variants have been discovered or constructed by biologists, and Cas9 nucleases and single guide RNAs（sgRNA） are the major components of the CRISPR-Cas9 system. These Cas9 systems have been extensively applied for identifying therapeutic targets, identifying gene functions, generating animal models, and developing gene therapies.Moreover, CRISPR-Cas9 systems have been used to partially or completely alleviate disease symptoms by mutating or correcting related genes. However, the efficient transfer of CRISPR-Cas9 system into cells and target organs remains a challenge that affects the robust and precise genome editing activity. The current review focuses on delivery systems for Cas9 mRNA, Cas9 protein, or vectors encoding the Cas9 gene and corresponding sgRNA. Non-viral delivery of Cas9 appears to help Cas9 maintain its on-target effect and reduce off-target effects, and viral vectors for sgRNA and donor template can improve the efficacy of genome editing and homology-directed repair. Safe, efficient, and producible delivery systems will promote the application of CRISPR-Cas9 technology in human gene therapy.
11-5841/Q
genome editing, CRISPR, Cas9, viral vector, non-viral vector, gene therapy
ObjectType-Article-1
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ObjectType-Feature-2
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ISSN:1674-7305
1869-1889
1869-1889
DOI:10.1007/s11427-017-9033-0