Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine , Baltimore, MD, USA.
Institute for Nanobiotechnology, Johns Hopkins University , Baltimore, MD, 21218, USA.
Expert Opin Drug Deliv. 2020 Oct;17(10):1395-1410. doi: 10.1080/17425247.2020.1796628. Epub 2020 Jul 31.
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 and .
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.
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.
基因传递技术正在为越来越多的生物医学应用而开发,传递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)作为一种合成的、可生物降解的聚合物,已成为一种领先的基因传递载体,已被用于多种应用。
本文综述了 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缺点。还总结了 PBAE 用于改善其他药物传递载体性能的用途。
PBAE 被设计为具有多种理想的基因传递特性,包括可逆正电荷,这有助于与核酸结合并赋予其高缓冲能力,以及在温和条件下快速降解。然而,其一些特性也导致纳米颗粒在生理环境中的不稳定性和低转染效率。PBAE 易于化学修饰以及与其他材料非共价混合,这使得它们能够专门定制以克服各种应用的传递障碍。