Nanotechnology-enabled gene delivery for cancer and other genetic diseases

被引:11
|
作者
Jiang, Tong [1 ]
Gonzalez, Karina Marie [1 ]
Cordova, Leyla Estrella [1 ]
Lu, Jianqin [1 ,2 ,3 ,4 ,5 ]
机构
[1] Univ Arizona, Skaggs Pharmaceut Sci Ctr, Dept Pharmacol & Toxicol, R Ken Coit Coll Pharm, Tucson, AZ USA
[2] NCI Designated Univ Arizona Comprehens Canc Ctr, Tucson, AZ USA
[3] Univ Arizona, BIO5 Inst, Tucson, AZ USA
[4] Univ Arizona, Southwest Environm Hlth Sci Ctr, Tucson, AZ USA
[5] Univ Arizona, Skaggs Pharmaceut Sci Ctr 422, Dept Pharmacol & Toxicol, R Ken Coit Coll Pharm,Pharmaceut & Pharmacokinet T, Tucson, AZ 85721 USA
基金
美国国家卫生研究院;
关键词
Gene therapy; nanotechnology; drug delivery system; non-viral vectors; viral vectors; GENOME-EDITING TECHNOLOGIES; BLOOD-BRAIN-BARRIER; RNA INTERFERENCE; IN-VIVO; SIRNA DELIVERY; DRUG-DELIVERY; TRANSCRIPTIONAL ACTIVATION; ANTISENSE OLIGONUCLEOTIDE; INTRACELLULAR DELIVERY; CHEMICAL-MODIFICATIONS;
D O I
10.1080/17425247.2023.2200246
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Introduction Despite gene therapy is ideal for genetic abnormality-related diseases, the easy degradation, poor targeting, and inefficiency in entering targeted cells are plaguing the effective delivery of gene therapy. Viral and non-viral vectors have been used for delivering gene therapeutics in vivo by safeguarding nucleic acid agents to target cells and to reach the specific intracellular location. A variety of nanotechnology-enabled safe and efficient systems have been successfully developed to improve the targeting ability for effective therapeutic delivery of genetic drugs.Areas covered In this review, we outline the multiple biological barriers associated with gene delivery process, and highlight recent advances to gene therapy strategy in vivo, including gene correction, gene silencing, gene activation and genome editing. We point out current developments and challenges exist of non-viral and viral vector systems in association with chemical and physical gene delivery technologies and their potential for the future.Expert opinion This review focuses on the opportunities and challenges to various gene therapy strategy, with specific emphasis on overcoming the challenges through the development of biocompatibility and smart gene vectors for potential clinical application.
引用
收藏
页码:523 / 540
页数:18
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