Synthetic Vehicles for Encapsulation and Delivery of CRISPR/Cas9 Gene Editing Machinery

被引：23
|
作者：
Carboni, Valentina [1 ]
Maaliki, Carine [1 ]
Alyami, Mram [1 ]
Alsaiari, Shahad [1 ]
Khashab, Niveen [1 ]
机构：
[1] KAUST, Adv Membranes & Porous Mat Ctr, Smart Hybrid Mat Lab SHMs, Thuwal 239556900, Saudi Arabia
来源：
ADVANCED THERAPEUTICS | 2019年 / 2卷 / 04期
关键词：
CRISPR/Cas9; delivery; gene editing; hybrid materials; nonviral vectors; organic materials; METAL-ORGANIC FRAMEWORKS; LIPID NANOPARTICLE FORMULATIONS; IN-VIVO; GOLD NANOPARTICLES; DRUG-DELIVERY; COMBINATORIAL LIBRARY; NONVIRAL DELIVERY; CO-DELIVERY; INTRACELLULAR DELIVERY; MAGNETIC NANOPARTICLES;
D O I：
10.1002/adtp.201800085
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
Clustered regularly interspaced short palindromic repeat (CRISPR) and CRISPR-associated 9 (Cas-9) technology holds tremendous potential as a gene editing tool. Different strategies have been adopted for in vitro and in vivo delivery of CRISPR/Cas9, including both viral and non-viral. The possibility of tailoring properties of nanosized systems makes the molecular design of self-assembled non-viral delivery systems based on organic (lipids and polymers) and hybrid (zeolitic imidazolate frameworks, ZIF and gold nanoparticles) materials of a great interest in CRISPR/Cas9 delivery. This review highlights the progress and challenges of organic and hybrid CRISPR/Cas9 delivery vehicles.
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页数：9
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