Lipid- and polymer-based nanoparticle systems for the delivery of CRISPR/Cas9

被引：34
作者：
Ashok, Bhaargavi [1 ]
Peppas, Nicholas A. [1 ,2 ,3 ,4 ,5 ]
Wechsler, Marissa E. [6 ]
机构：
[1] Univ Texas Austin, Dept Chem Engn, Austin, TX 78712 USA
[2] Univ Texas Austin, Inst Biomat Drug Delivery & Regenerat Med, Austin, TX 78712 USA
[3] Univ Texas Austin, Dept Biomed Engn, Austin, TX 78712 USA
[4] Univ Texas Austin, Coll Pharm, Div Mol Pharmaceut & Drug Delivery, Austin, TX 78712 USA
[5] Univ Texas Austin, Dell Med Sch, Dept Surg & Perioperat Care, Austin, TX 78712 USA
[6] Univ Texas San Antonio, Dept Biomed Engn & Chem Engn, San Antonio, TX 78249 USA
来源：
JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY | 2021年 / 65卷
基金：
美国国家卫生研究院;
关键词：
Hydrogels; Nanoparticles; CRISPR; Genome editing; IN-VIVO; TARGETED DELIVERY; MESSENGER-RNA; T-CELLS; GENE; CARRIERS; CANCER; VITRO; DNA; CRISPR-CAS9;
D O I：
10.1016/j.jddst.2021.102728
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
The discovery of clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas) genome editing systems and their applications in human health and medicine has heralded a new era of biotechnology. However, the delivery of CRISPR therapeutics is arguably the most difficult barrier to overcome for translation to in vivo clinical administration. Appropriate delivery methods are required to efficiently and selectively transport all gene editing components to specific target cells and tissues of interest, while minimizing off-target effects. To overcome this challenge, we discuss and critique nanoparticle delivery strategies, focusing on the use of lipid-based and polymeric-based matrices herein.
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页数：13
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