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细胞穿透肽,基因治疗的新型载体。

Cell Penetrating Peptides, Novel Vectors for Gene Therapy.

作者信息

Taylor Rebecca E, Zahid Maliha

机构信息

Mechanical Engineering, Biomedical Engineering and Electrical and Computer Engineering, Carnegie Mellon University, Pittsburgh, PA 15213, USA.

Department of Developmental Biology, University of Pittsburgh School of Medicine, Pittsburgh, PA 15201, USA.

出版信息

Pharmaceutics. 2020 Mar 3;12(3):225. doi: 10.3390/pharmaceutics12030225.

Abstract

Cell penetrating peptides (CPPs), also known as protein transduction domains (PTDs), first identified ~25 years ago, are small, 6-30 amino acid long, synthetic, or naturally occurring peptides, able to carry variety of cargoes across the cellular membranes in an intact, functional form. Since their initial description and characterization, the field of cell penetrating peptides as vectors has exploded. The cargoes they can deliver range from other small peptides, full-length proteins, nucleic acids including RNA and DNA, liposomes, nanoparticles, and viral particles as well as radioisotopes and other fluorescent probes for imaging purposes. In this review, we will focus briefly on their history, classification system, and mechanism of transduction followed by a summary of the existing literature on use of CPPs as gene delivery vectors either in the form of modified viruses, plasmid DNA, small interfering RNA, oligonucleotides, full-length genes, DNA origami or peptide nucleic acids.

摘要

细胞穿透肽(CPPs),也被称为蛋白质转导结构域(PTDs),约25年前首次被发现,是一种由6至30个氨基酸组成的小型合成或天然存在的肽,能够以完整、功能性的形式携带各种货物穿过细胞膜。自其最初被描述和表征以来,作为载体的细胞穿透肽领域得到了迅猛发展。它们能够递送的货物范围广泛,包括其他小肽、全长蛋白质、核酸(包括RNA和DNA)、脂质体、纳米颗粒和病毒颗粒,以及用于成像目的的放射性同位素和其他荧光探针。在本综述中,我们将简要关注其历史、分类系统和转导机制,随后总结现有文献中关于将CPPs用作基因递送载体的研究,这些载体形式包括修饰病毒、质粒DNA、小干扰RNA、寡核苷酸、全长基因、DNA折纸或肽核酸。

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