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作为基因载体的生物相容性和可生物降解纳米粒子的最新进展。

Recent advances on biocompatible and biodegradable nanoparticles as gene carriers.

机构信息

a Research Center for Pharmaceutical Nanotechnology , Tabriz University of Medical Sciences , Tabriz , Iran.

b Department of Biotechnology , Higher Education Institute of Rab-Rashid , Tabriz , Iran.

出版信息

Expert Opin Biol Ther. 2016 Jun;16(6):771-85. doi: 10.1517/14712598.2016.1169269. Epub 2016 Apr 4.

Abstract

INTRODUCTION

Gene therapy mainly depends on the use of appropriate delivery vehicles with no induction of immune responses and toxicity. The limitations of viral gene carriers such as induction of immunogenicity, random integration in the genome of the host, limitations in the size, has led to a movement toward non-viral systems with much safer properties. Biodegradable and biocompatible polymeric nanocarriers due to several unique properties such as excellent biocompatibility, prolonged gene circulation time, prevented gene degradation, passive targeting by using the enhanced permeability and retention (EPR) effect, and possibility of modulating polymers structure to obtain desirable therapeutic efficacy, are among the most promising systems for gene delivery. However, biodegradable gene delivery systems have some limitations such as inadequate stability and slow release of therapeutics which have to be overcome. Thus, a variety of advanced functional biodegradable delivery systems with more efficient gene delivery activity has recently been introduced.

AREAS COVERED

This review summarizes different aspects of biodegradable and biocompatible nano carriers including formulation, mechanism of intracellular uptake, various potential applications of biodegradable nanoparticles and finally recent studies on the therapeutic efficacy of these nanoparticles in sustained delivery of genes.

EXPERT OPINION

Biocompatible and biodegradable polymers will play a necessary and important role in developing new and safe carriers for oligonucleotide delivery. More working and the development of optimized polymers will reveal more their efficacy in the treatment of patients via helping in better gene therapy.

摘要

简介

基因治疗主要依赖于使用适当的载体,而不会引起免疫反应和毒性。病毒基因载体的局限性,如诱导免疫原性、在宿主基因组中的随机整合、大小限制等,促使人们转向非病毒系统,这些系统具有更安全的特性。可生物降解和生物相容的聚合物纳米载体由于具有许多独特的性质,如优异的生物相容性、延长基因循环时间、防止基因降解、通过增强的通透性和保留(EPR)效应被动靶向,以及调节聚合物结构以获得理想的治疗效果的可能性,是最有前途的基因传递系统之一。然而,可生物降解的基因传递系统存在一些局限性,如稳定性不足和治疗药物释放缓慢,这些局限性必须克服。因此,最近已经引入了各种具有更高基因传递活性的先进功能性可生物降解传递系统。

涵盖领域

这篇综述总结了可生物降解和生物相容的纳米载体的不同方面,包括制剂、细胞内摄取机制、可生物降解纳米粒子的各种潜在应用,以及最近这些纳米粒子在基因持续传递中的治疗效果的研究。

专家意见

生物相容和可生物降解的聚合物将在开发新的和安全的寡核苷酸传递载体方面发挥必要和重要的作用。更多的工作和优化聚合物的开发将揭示它们在通过帮助更好的基因治疗来治疗患者方面的疗效。

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