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新型聚乙烯亚胺衍生纳米颗粒用于体内基因传递。

Novel polyethylenimine-derived nanoparticles for in vivo gene delivery.

机构信息

CSIR-Indian Institute of Toxicology Research, M.G. Marg, Lucknow, 226 001, India.

出版信息

Expert Opin Drug Deliv. 2013 Feb;10(2):215-28. doi: 10.1517/17425247.2013.744964. Epub 2012 Dec 19.

Abstract

INTRODUCTION

Branched and linear polyethylenimines (PEIs) are cationic polymers that have been used to deliver nucleic acids both in vitro and in vivo. Owing to the high cationic charge, the branched polymers exhibit high transfection efficiency, and particularly PEI of molecular weight 25 kDa is considered as a gold standard in gene delivery. These polymers have been extensively studied and modified with different ligands so as to achieve the targeted delivery.

AREAS COVERED

The application of PEI in vivo promises to take the polymer-based vector to the next level wherein it can undergo clinical trials and subsequently could be used for delivery of therapeutics in humans. This review focuses on the various recent developments that have been made in the field of PEI-based delivery vectors for delivery of therapeutics in vivo.

EXPERT OPINION

The efficacy of PEI-based delivery vectors in vivo is significantly high and animal studies demonstrate that such systems have a potential in humans. However, we feel that though PEI is a promising vector, further studies involving PEI in animal models are needed so as to get a detailed toxicity profile of these vectors. Also, it is imperative that the vector reaches the specific organ causing little or no undesirable effects to other organs.

摘要

简介

支化和线性聚乙烯亚胺(PEI)是阳离子聚合物,已被用于体外和体内递送核酸。由于高正电荷,支化聚合物表现出高转染效率,特别是分子量为 25 kDa 的 PEI 被认为是基因传递的金标准。这些聚合物已经经过广泛研究并与不同的配体进行了修饰,以实现靶向递送。

涵盖领域

PEI 在体内的应用有望将基于聚合物的载体提升到一个新的水平,使其能够进行临床试验,随后可用于向人类输送治疗药物。本综述重点介绍了在基于 PEI 的治疗药物体内递送载体领域的最新进展。

专家意见

基于 PEI 的递送载体在体内的疗效非常高,动物研究表明,这些系统在人类中具有潜力。然而,我们认为,尽管 PEI 是一种很有前途的载体,但仍需要在动物模型中进一步研究 PEI,以获得这些载体的详细毒性概况。此外,至关重要的是,载体到达特定器官,对其他器官几乎没有或没有不良影响。

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