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用于遗传性疾病基因治疗的腺病毒载体

Adenoviral Vectors for Gene Therapy of Hereditary Diseases.

作者信息

Muravyeva Anna, Smirnikhina Svetlana

机构信息

Laboratory of Genome Editing, Research Centre for Medical Genetics, Moskvorechye, 1, 115522 Moscow, Russia.

出版信息

Biology (Basel). 2024 Dec 16;13(12):1052. doi: 10.3390/biology13121052.

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.

摘要

腺病毒载体(AdV)因其广泛的嗜性、高容量和高转导效率,是基因治疗的有效载体,这使得它们被积极用作溶瘤载体和用于制备载体疫苗。然而,尽管腺病毒载体有诸多优点,但在遗传性疾病的基因治疗中尚未找到用武之地。本综述概述了腺病毒载体、其特点以及在单基因疾病的基因替代治疗中使用它们的临床试验,并分析了这些研究失败的原因。此外,还讨论了目前关于修饰腺病毒载体以降低免疫反应和靶向递送的研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cd71/11673936/c9dfd3634306/biology-13-01052-g001.jpg

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