Advances on nonviral vectors of CRISPR/Cas9 system for genome editing
10.16438/j.0513-4870.2020-0484
- VernacularTitle:非病毒载体递送CRISPR/Cas9系统的研究进展
- Author:
Yu WANG
1
;
Gui HUANG
1
;
Han YANG
2
;
Xue-nong ZHANG
1
Author Information
1. College of Pharmaceutical Sciences, Soochow University, Suzhou 215123, China
2. Jiangsu Coastal Pharmaceutical Co., Ltd., Suzhou 215215, China
- Publication Type:Research Article
- Keywords:
genome editing;
CRISPR/Cas9;
nonviral vector;
liposome;
polymer
- From:
Acta Pharmaceutica Sinica
2020;55(11):2606-2617
- CountryChina
- Language:Chinese
-
Abstract:
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR associated protein system exerts genome editing effect through cleaving DNA double strands using RNA-guided endonuclease. Double-strand breaks were repaired via homology directed repair (HDR) or nonhomologous end joining (NHEJ), accompanied by insertions, deletions or replacements into the genome. As a powerful tool, CRISPR/Cas system has provided tremendous convenience for basic researches and may pave the path to treat genetic diseases and cancers. Genome editing could be achieved only when both CRISPR RNA and Cas protein are delivered into nucleus of target cell. Compared with physical and viral delivery, nonviral delivery of CRISPR/Cas system possesses unique advantages in terms of safety, loading capacity and preparation. Hence, many researchers have devoted themselves to the development of nonviral vectors with high delivery efficiency which is important for the application and translation of the promising technology. Advances on cationic liposomes, lipid like nanoparticles, cationic polymers, AuNPs, vesicles, polypeptides, proteins and so on have been made. We will give a brief introduction to the mechanism of CRISPR/Cas9, problems faced by nonviral delivery of CRISPR/Cas9 system in forms of plasmid, mRNA and protein; examples of non-viral vectors, hoping to give some hints on design of safe and efficient nonviral vectors for genome editing.
