人类病毒样蛋白:对基因疗法的影响。

IF 3.8 4区 医学 Q2 GENETICS & HEREDITY
Aya Al Othman, Anna Polyanskaya, Mikhail Durymanov
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引用次数: 0

摘要

通过对哺乳动物基因组的分析,发现了大量源自转座子或病毒的 DNA 序列。其中一些元件编码功能蛋白,在进化过程中被重新利用,在某些组织中发挥重要的生理作用。一些人类病毒样蛋白,如 Peg10 和 Arc/Arg3.1,在结构上与 Gag 逆转录病毒蛋白非常相似,而另一些蛋白,如 syncytins-1 和-2,则类似于包膜病毒蛋白。近年来,这些蛋白显然可用于生物工程 "人性化 "的囊膜颗粒,以定向传递 mRNA。实现这一想法可为基因治疗提供高效的类病毒颗粒,并解决病毒载体的免疫原性问题。本综述概述了研究最多的病毒或转座子来源的人类蛋白质,并重点介绍了它们的生物学功能。此外,还讨论了利用这些蛋白质进行 mRNA 靶向传递的最新进展及其临床应用前景。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Human Virus-Like Proteins: Implications for Gene Therapy.

An analysis of mammalian genomes has revealed a significant number of DNA sequences with transposon or viral origin. Some of these elements encode functional proteins, repurposed during evolution to play significant physiological roles in certain tissues. Some human virus-like proteins, such as Peg10 and Arc/Arg3.1, structurally demonstrate significant similarity with Gag retroviral proteins, while others, like syncytins-1 and -2, resemble envelope viral proteins. In recent years, it has become clear that these proteins can be exploited for bioengineering 'humanized' capsid particles aimed at targeted mRNA delivery. Realizing this idea could provide efficient virus-like particles for gene therapy and address the problem of viral vector immunogenicity. This review provides an overview of the most-studied human proteins of viral or transposon origin and highlights their biological functions. Additionally, recent advances in exploiting these proteins for targeted mRNA delivery and prospects for their clinical application are discussed.

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来源期刊
Current gene therapy
Current gene therapy 医学-遗传学
CiteScore
6.70
自引率
2.80%
发文量
46
期刊介绍: Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases. Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.
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