Combinatorial library of chalcogen-containing lipidoids for intracellular delivery of genome-editing proteins

被引：66

作者：

Li, Yamin ^{[1
]}

Yang, Tao ^{[1
,2
]}

Yu, Yingjie ^{[1
]}

Shi, Nicola ^{[1
]}

Yang, Liu ^{[1
]}

Glass, Zachary ^{[1
]}

Bolinger, Justin ^{[1
]}

Finkel, Isaac James ^{[1
]}

Li, Wenhan ^{[1
]}

Xu, Qiaobing ^{[1
]}

机构：

[1] Tufts Univ, Dept Biomed Engn, Medford, MA 02155 USA

[2] Sichuan Univ, West China Hosp Stomatol, Natl Clin Res Ctr Oral Dis, State Key Lab Oral Dis, Chengdu 610065, Sichuan, Peoples R China

来源：

BIOMATERIALS | 2018年 / 178卷

基金：

美国国家科学基金会;

关键词：

Lipidoids; Protein delivery; CRISPR/Cas9; Genome editing; IN-VITRO; CAS9; RIBONUCLEOPROTEIN; EFFICIENT DELIVERY; NONVIRAL DELIVERY; NANOPARTICLES; CRISPR-CAS9; NANOMATERIALS; CHALLENGES; COPOLYMER; SYSTEM;

D O I：

10.1016/j.biomaterials.2018.03.011

中图分类号：

R318 [生物医学工程];

学科分类号：

0831 ;

摘要：

Protein based therapeutics with high specificities and low off-target effects are used for transient and accurate manipulation of cell functions. However, developing safe and efficient carriers for intracellular delivery of active therapeutic proteins is a long-standing challenge. Here we report a combinatorial library of chalcogen (O, S, Se) containing lipidoid nanoparticles (LNPs) as efficient nanocarriers for intracellular delivery of negatively supercharged Cre recombinase ((-30)GFP-Cre) and anionic Cas9:single-guide RNA (Cas9:sgRNA) ribonucleoprotein (RNP) for genome editing. The structure-activity relationship between the lipidoids and intracellular protein delivery efficiencies was explored and it was demonstrated that the newly developed LNPs are effective for gene recombination in vivo. (C) 2018 Elsevier Ltd. All rights reserved.

引用

页码：652 / 662

页数：11

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