Development of delivery strategies for CRISPR-Cas9 genome editing

被引:29
作者
Liu, Qi [1 ]
Yang, Jianhui [1 ]
Xing, Yumeng [1 ]
Zhao, Yu [2 ]
Liu, Yang [3 ]
机构
[1] Anhui Med Univ, Sch Pharm, Hefei 230032, Peoples R China
[2] Tufts Univ, Dept Biomed Engn, Medford, MA USA
[3] Nankai Univ, Coll Chem, Minist Educ, State Key Lab Med Chem Biol ,Key Lab Funct Polymer, Tianjin 300071, Peoples R China
来源
BMEMAT | 2023年 / 1卷 / 03期
基金
中国国家自然科学基金;
关键词
CRISPR-Cas9; delivery strategy; genome editing; non-viral nanocarriers; stimulating-responsive nanocarriers; MESOPOROUS SILICA NANOPARTICLES; SMART DRUG-DELIVERY; TARGETED DELIVERY; HUMAN-CELLS; CAS9; RIBONUCLEOPROTEIN; NONVIRAL VECTORS; VIRAL VECTORS; NUCLEIC-ACIDS; VIVO; CRISPR/CAS9;
D O I
10.1002/bmm2.12025
中图分类号
TB3 [工程材料学];
学科分类号
0805 ; 080502 ;
摘要
The clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-related protein 9 (Cas9) genome editing system has attracted much attention due to its powerful genome editing capacity. However, CRISPR-Cas9 components are easily degraded by acids, enzymes, and other substances in the body fluids after entering the organism, thus efficiently delivering the CRISPR-Cas9 system into targeted organs or cells has been a central theme for promoting the application of CRISPR-Cas9 technology. Although several physical methods and viral vectors have been developed for CRISPR-Cas9 delivery, their clinical application still suffers from disadvantages, such as the risks of mutagenesis, cell damage, and poor specificity. As an alternative, non-viral nanocarriers hold great promise for circumventing these challenges. Furthermore, with aim to realize more efficient and precise genome editing and reduce the undesirable side effects, stimuli-responsive nanocarriers are designed for the spatiotemporal CRISPR-Cas9 delivery in responsive to various stimuli. In this review, we will summarize the recent progress in delivery strategies for CRISPR-Cas9 genome editing. The mechanisms and advantages of these strategies were reviewed, providing a comprehensive review of the rational design of materials and techniques for efficient and precise genome editing. At last, the potential challenges of current CRISPR-Cas9 delivery are discussed. The development of delivery strategies of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-related protein 9 (Cas9) system has been a central theme for promoting the application of CRISPR-Cas9 technology. In this review, we summarize the recent progress in delivery strategies for effective CRISPR-Cas9 genome editing and discuss the potential challenges of current CRISPR-Cas9 delivery. image
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页数:30
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