Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing

Tang, Hao1; Zhao, Xiaohui1; Jiang, Xingyu1,2,3,4

2021

10.1016/j.addr.2020.03.001

ADVANCED DRUG DELIVERY REVIEWS   影响因子和分区

0169-409X

1872-8294

The clustered regularly interspaced short palindromic repeat (CRISPR) has great potential to revolutionize biomedical research and disease therapy. The specific and efficient genome editing strongly depends on high efficiency of delivery of the CRISPR payloads. However, optimization of CRISPR delivery vehicles still remains a major obstacle. Recently, various non-viral vectors have been utilized to deliver CRISPR tools. Many of these vectors have multi-layer structures assembled. In this review, we will introduce the development of CRISPR-Cas9 systems and their general therapeutic applications by summarizing current CRISPR-Cas9 based clinical trials. We will highlight the multi-layer nanoparticles (NPs) that have been developed to deliver CRISPR cargos in vitro and in vivo for various purposes, as well the potential building blocks of multi-layer NPs. We will also discuss the challenges in making the CRISPR tools into viable pharmaceutical products and provide potential solutions on efficiency and biosafety issues. (c) 2020 Published by Elsevier B.V.

Genome editing CRISPR-Cas9 Non-viral vectors Multi-layer NPs Clinical transformation

EI ; SCI

英语

第一 ; 通讯

National Key R&D Program of China["2018YFA0902600","2017YFA0205901"] ; National Natural Science Foundation of China[21535001,81730051,21761142006] ; Chinese Academy of Sciences["QYZDJ-SSW-SLH039","121D11KYSB20170026","XDA16020902"]

Pharmacology & Pharmacy

Pharmacology & Pharmacy

WOS:000612867300004

ELSEVIER

20201108305255

Disease control ; Efficiency ; Genes

Biological Materials and Tissue Engineering:461.2 ; Nanotechnology:761 ; Production Engineering:913.1 ; Solid State Physics:933

PHARMACOLOGY & TOXICOLOGY

Web of Science

1.Southern Univ Sci & Technol, Dept Biomed Engn, 1088 Xueyuan Rd, Shenzhen 518055, Guangdong, Peoples R China
2.CAS Ctr Excellence Nanosci, Beijing Engn Res Ctr BioNanotechnol, Natl Ctr NanoSci & Technol, 11 Zhongguancun Beiyitiao, Beijing 100190, Peoples R China
3.CAS Ctr Excellence Nanosci, CAS Key Lab Biomed Effects Nanomat & Nanosafety, Natl Ctr NanoSci & Technol, 11 Zhongguancun Beiyitiao, Beijing 100190, Peoples R China
4.Univ Chinese Acad Sci, 19 A Yuquan Rd, Beijing 100049, Peoples R China

Tang, Hao,Zhao, Xiaohui,Jiang, Xingyu. Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing[J]. ADVANCED DRUG DELIVERY REVIEWS,2021,168:55-78.

Tang, Hao,Zhao, Xiaohui,&Jiang, Xingyu.(2021).Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing.ADVANCED DRUG DELIVERY REVIEWS,168,55-78.

Tang, Hao,et al."Synthetic multi-layer nanoparticles for CRISPR-Cas9 genome editing".ADVANCED DRUG DELIVERY REVIEWS 168(2021):55-78.