Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

被引：81

作者：

Givens, Brittany E. ^{[1
,2
]}

Naguib, Youssef W. ^{[1
,3
]}

Geary, Sean M. ^{[1
]}

Devor, Eric J. ^{[4
]}

Salem, Aliasger K. ^{[1
,2
]}

机构：

[1] Univ Iowa, Coll Pharm, Div Pharmaceut & Translat Therapeut, Iowa City, IA 52242 USA

[2] Univ Iowa, Coll Pharm, Dept Chem & Biochem Engn, Iowa City, IA 52242 USA

[3] Minia Univ, Fac Pharm, Dept Pharmaceut, Al Minya 61519, Egypt

[4] Univ Iowa, Dept Obstet & Gynecol, Carver Coll Med, Iowa City, IA 52242 USA

来源：

AAPS JOURNAL | 2018年 / 20卷 / 06期

基金：

美国国家卫生研究院;

关键词：

CRISPR; Cas9; gene editing; gene delivery; nanoparticle; CELL-PENETRATING PEPTIDES; SOLID LIPID NANOPARTICLES; FIBROBLAST-GROWTH-FACTOR; IN-VIVO; MESSENGER-RNA; GENE DELIVERY; NONVIRAL VECTORS; SIRNA DELIVERY; BIODEGRADABLE MICROPARTICLES; TRANSFECTION EFFICIENCY;

D O I：

10.1208/s12248-018-0267-9

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

引用

页数：22

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