期刊
NANOSCALE
卷 12, 期 41, 页码 21001-21014出版社
ROYAL SOC CHEMISTRY
DOI: 10.1039/d0nr05452f
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资金
- National Key Research and Development Program of China [2016YFA0201400]
- State Key Program of National Natural Science of China [81930047]
- Projects of International Cooperation and Exchanges NSFC-PSF [31961143003]
- National Project for Research and Development of Major Scientific Instruments [81727803]
- Beijing Natural Science Foundation, Haidian, Original Innovation Joint Fund [17L20170]
- Foundation for Innovative Research Groups of the National Natural Science Foundation of China [81421004]
CRISPR-Cas9, the breakthrough genome-editing technology, has emerged as a promising tool to prevent and cure various diseases. The efficient genome editing technology strongly relies on the specific and effective delivery of CRISPR/Cas9 cargos. However, the lack of a safe, specific, and efficient non-viral delivery system for in vivo genome editing remains a major limit for its clinical translation. In this review, we will first briefly introduce the working mechanism of CRISPR/Cas9 and the patterns of CRISPR/Cas9 delivery. Furthermore, the physiological obstacles for the delivery process in vivo are elaborated. Finally, the key considerations will be deeply discussed in designing non-viral nanovectors for therapeutic CRISPR/Cas9 delivery in vivo, including the effective encapsulation of large-size macromolecules, targeting specific tissues and cells, efficient endosomal escape and safety concerns of the vector systems, in the hope of inviting more comprehensive studies on the development of safe, specific, and efficient non-viral nanovectors for delivering a CRISPR/Cas9 system.
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