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Delivery of CRISPR/Cas9 for therapeutic genome editing
Abstract
The clustered, regularly‐interspaced, short palindromic repeat (CRISPR)‐associated nuclease 9 (CRISPR/Cas9) is emerging as a promising genome‐editing tool for treating diseases in a precise way, and has been applied to a wide range of research in the areas of biology, genetics, and medicine. Delivery of therapeutic genome‐editing agents provides a promising platform for the treatment of genetic disorders. Although viral vectors are widely used to deliver CRISPR/Cas9 elements with high efficiency, they suffer from several drawbacks, such as mutagenesis, immunogenicity, and off‐target effects. Recently, non‐viral vectors have emerged as another class of delivery carriers in terms of their safety, simplicity, and flexibility. In this review, we discuss the modes of CRISPR/Cas9 delivery, the barriers to the delivery process and the application of CRISPR/Cas9 system for the treatment of genetic disorders. We also highlight several representative types of non‐viral vectors, including polymers, liposomes, cell‐penetrating peptides, and other synthetic vectors, for the therapeutic delivery of CRISPR/Cas9 system. The applications of CRISPR/Cas9 in treating genetic disorders mediated by the non‐viral vectors are also discussed.
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“The positively charged liposomes encapsulated with negatively charged nucleic acids thereby facilitate the fusion of the complex across the cell membrane into cells.59”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [59]
Citations
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Sorted by citations
Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing
Mengstie, M. A., & Wondimu, B. Z. (2021) · Biologics: Targets and Therapy · doi:10.2147/btt.s326422 · Cited by 478
Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
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Exosome/Liposome-like Nanoparticles: New Carriers for CRISPR Genome Editing in Plants
Alghuthaymi, M. A., Ahmad, A., Khan, Z., Khan, S. H., Ahmed, F. K., Faiz, S., Nepovimova, E., Kuča, K., & Abd-Elsalam, K. A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22147456 · Cited by 83
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Metadata
- Authors
- Xu, X., Wan, T., Xin, H., Li, D., Pan, H., Wu, J., & Ping, Y.
- Affiliations
- College of Pharmaceutical Sciences Zhejiang University 866 Yuhangtang Road Hangzhou 310058 China
- Department of Medical Oncology Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University Hangzhou 310016 China
- School of Biomedical Engineering Sun Yat‐sen University Guangzhou 510006 China
- Venue
- The Journal of Gene Medicine
- Publisher
- Wiley
- Published
- 2019-07
- Volume
- 21
- Issue
- 7
- ISSN
- 1099-498X
- 1521-2254
- Cited by
- 134
- References
- 30
- Language
- en
- Funders
- Ministry of Science and Technology of the People's Republic of China (2018ZX10301402)
- Fundamental Research Funds for the Central Universities (2018XZZX001‐14)
- National Natural Science Foundation of China (81872807)
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