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用于传染病基因治疗的细胞内抗体

Intracellular antibodies (intrabodies) for gene therapy of infectious diseases.

作者信息

Rondon I J, Marasco W A

机构信息

Division of Human Retrovirology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, Massachusetts 02115, USA.

出版信息

Annu Rev Microbiol. 1997;51:257-83. doi: 10.1146/annurev.micro.51.1.257.

Abstract

Intracellular antibodies (intrabodies) represent a new class of neutralizing molecules with a potential use in gene therapy. Intrabodies are engineered single-chain antibodies in which the variable domain of the heavy chain is joined to the variable domain of the light chain through a peptide linker, preserving the affinity of the parent antibody. Intrabodies are expressed inside cells and directed to different subcellular compartments where they can exert their function more effectively. The effects of intrabodies have been investigated using structural, regulatory, and enzymatic proteins of the human immunodeficiency virus (HIV-1) as targets. These intrabodies have demonstrated their versatility by controlling early as well as late events of the viral life cycle. In this article, we review studies of the use of intrabodies as research tools and therapeutic agents against HIV-1.

摘要

细胞内抗体(intrabodies)代表了一类新型的中和分子,在基因治疗中具有潜在应用价值。细胞内抗体是经过工程改造的单链抗体,其中重链可变区通过肽接头与轻链可变区相连,保留了亲本抗体的亲和力。细胞内抗体在细胞内表达,并靶向不同的亚细胞区室,在那里它们可以更有效地发挥功能。已将人类免疫缺陷病毒(HIV-1)的结构、调节和酶蛋白作为靶点,研究了细胞内抗体的作用。这些细胞内抗体通过控制病毒生命周期的早期和晚期事件,展示了它们的多功能性。在本文中,我们综述了将细胞内抗体用作针对HIV-1的研究工具和治疗剂的研究。

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