Spatiotemporal Delivery of CRISPR/Cas9 Genome Editing Machinery Using Stimuli-Responsive Vehicles
Weiqi Cai
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorTianli Luo
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorProf. Lanqun Mao
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorCorresponding Author
Prof. Ming Wang
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorWeiqi Cai
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorTianli Luo
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorProf. Lanqun Mao
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorCorresponding Author
Prof. Ming Wang
Beijing National Laboratory for Molecular Science, CAS Key Laboratory of Analytical Chemistry for Living Biosystems, Institute of Chemistry, Chinese Academy of Sciences (ICCAS), No. 2, North first street, Zhongguancun, Beijing, 100190 China
University of Chinese Academy of Sciences, No.19 (A) Yuquan Road, Shijingshan District, China
Search for more papers by this authorGraphical Abstract
Spatiotemporal delivery of CRISPR/Cas9 genome editing machinery is of great importance for developing chemical biology tools for manipulating cellular DNA sequences to treat genetic disorders. This Minireview provides an overview of synthetic approaches and specific chemistries that have been used to develop stimuli-responsive vehicles for CRISPR/Cas9 delivery and genome editing in a controlled manner.
Abstract
Recent innovations in genome editing have enabled the precise manipulation of the genetic information of mammalians, and benefitted the development of next-generation gene therapy. Despite these advances, several barriers to the clinical translation of genome editing remain, including the intracellular delivery of genome editing machinery, and the risk of off-target editing effect. Here, we review the recent advance of spatiotemporal delivery of CRISPR/Cas9 genome editing machinery, which is composed of programmable Cas9 nuclease and a single-guide RNA (sgRNA) using stimuli-responsive nanoparticles. We discuss the specific chemistries that have been used for controlled Cas9/sgRNA delivery and intracellular release in the presence of endogenous or external signals. These methodologies can leverage biological signals found locally within disease cells, or exogenous signals administrated with spatiotemporal control, through which an improved genome editing could be achieved. We also discuss the future in exploiting these approaches for fundamental biomedical applications and therapeutic genome editing.
Conflict of interest
The authors declare no conflict of interest.
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