Adenoviral Vectors for Gene Therapy of Hereditary Diseases.

IF 3.6 3区 生物学 Q1 BIOLOGY
Anna Muravyeva, Svetlana Smirnikhina
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引用次数: 0

Abstract

Adenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines. However, despite their numerous advantages, AdVs have not yet found their place in gene therapy for hereditary diseases. This review provides an overview of AdVs, their features, and clinical trials using them for gene replacement therapy in monogenic diseases and analyzes the reasons for the failures of these studies. Additionally, current research on the modification of AdVs to reduce immune responses and target delivery is discussed.

用于遗传性疾病基因治疗的腺病毒载体。
腺病毒载体(Adenoviral vector, AdVs)因其广泛的趋向性、高容量和高转导效率而成为基因治疗的有效载体,被广泛用于溶瘤载体和载体疫苗的制备。然而,尽管AdVs有许多优点,但它还没有在遗传性疾病的基因治疗中找到自己的位置。本文综述了AdVs、AdVs的特点、用于单基因疾病基因替代治疗的临床试验,并分析了这些研究失败的原因。此外,还讨论了目前对AdVs进行修饰以减少免疫反应和靶向递送的研究进展。
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
Biology-Basel
Biology-Basel Biological Science-Biological Science
CiteScore
5.70
自引率
4.80%
发文量
1618
审稿时长
11 weeks
期刊介绍: Biology (ISSN 2079-7737) is an international, peer-reviewed, quick-refereeing open access journal of Biological Science published by MDPI online. It publishes reviews, research papers and communications in all areas of biology and at the interface of related disciplines. Our aim is to encourage scientists to publish their experimental and theoretical results in as much detail as possible. There is no restriction on the length of the papers. The full experimental details must be provided so that the results can be reproduced. Electronic files regarding the full details of the experimental procedure, if unable to be published in a normal way, can be deposited as supplementary material.
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