ABSTRACT

Introduction

Gene delivery technologies are being developed for an increasing number of biomedical applications, with delivery vehicles including viruses and non-viral materials. Among biomaterials used for non-viral gene delivery, poly(beta-amino ester)s (PBAEs), a class of synthetic, biodegradable polymers, have risen as a leading gene delivery vehicle that has been used for multiple applications in vitro and in vivo.

Areas covered

This review summarizes the key properties of PBAEs and their development, including a discussion of the advantages and disadvantages of PBAEs for gene delivery applications. The use of PBAEs to improve the properties of other drug delivery vehicles is also summarized.

Expert opinion

PBAEs are designed to have multiple characteristics that are ideal for gene delivery, including their reversible positive charge, which promotes binding to nucleic acids as well as imparting high buffering capacity, and their rapid degradability under mild conditions. Simultaneously, some of their properties also lead to nanoparticle instability and low transfection efficiency in physiological environments. The ease with which PBAEs can be chemically modified as well as non-covalently blended with other materials, however, allows them to be customized specifically to overcome delivery barriers for varied applications.

中文翻译：

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聚（β-氨基酯）作为基因传递载体：挑战和机遇。

 抽象的

 介绍

基因递送技术正在为越来越多的生物医学应用而开发，其递送载体包括病毒和非病毒材料。在用于非病毒基因传递的生物材料中，聚（β-氨基酯）（PBAE）是一类合成的、可生物降解的聚合物，已成为一种领先的基因传递载体，已在体外和体内多种应用。

 涵盖领域

本综述总结了 PBAE 的关键特性及其发展，包括讨论 PBAE 在基因传递应用中的优点和缺点。还总结了使用 PBAE 来改善其他药物输送载体的性能。

 专家意见

PBAE 被设计为具有多种非常适合基因传递的特性，包括其可逆的正电荷，可促进与核酸的结合并赋予高缓冲能力，以及在温和条件下的快速降解性。同时，它们的一些特性也导致纳米颗粒在生理环境中不稳定和转染效率低。然而，PBAE 可以轻松地进行化学改性以及与其他材料非共价混合，因此可以专门定制它们，以克服各种应用的输送障碍。