Nanotechnology-enabled gene delivery for cancer and other genetic diseases.

IF 5 2区 医学 Q1 PHARMACOLOGY & PHARMACY
Expert Opinion on Drug Delivery Pub Date : 2023-04-01 Epub Date: 2023-04-10 DOI:10.1080/17425247.2023.2200246
Tong Jiang, Karina Marie Gonzalez, Leyla Estrella Cordova, Jianqin Lu
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引用次数: 0

Abstract

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.

利用纳米技术传递基因,治疗癌症和其他遗传疾病。
导言:尽管基因疗法是治疗与基因异常有关的疾病的理想方法,但其易降解、靶向性差、进入靶细胞的效率低等问题却困扰着基因疗法的有效传递。病毒和非病毒载体通过保护核酸制剂进入靶细胞并到达特定的细胞内位置,已被用于体内递送基因治疗药物。目前已成功开发出多种安全高效的纳米技术系统,以提高基因药物的靶向能力,实现有效的治疗传递:在这篇综述中,我们概述了与基因递送过程相关的多种生物学障碍,并重点介绍了体内基因治疗策略的最新进展,包括基因校正、基因沉默、基因激活和基因组编辑。我们指出了非病毒和病毒载体系统以及化学和物理基因递送技术目前的发展和面临的挑战,以及它们在未来的潜力:本综述重点探讨了各种基因治疗策略所面临的机遇和挑战,特别强调通过开发生物相容性和智能基因载体来克服挑战,从而实现潜在的临床应用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
11.10
自引率
3.00%
发文量
104
审稿时长
3 months
期刊介绍: Expert Opinion on Drug Delivery (ISSN 1742-5247 [print], 1744-7593 [electronic]) is a MEDLINE-indexed, peer-reviewed, international journal publishing review articles covering all aspects of drug delivery research, from initial concept to potential therapeutic application and final relevance in clinical use. Each article is structured to incorporate the author’s own expert opinion on the scope for future development.
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