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聚合物囊泡用于蛋白质和核酸大分子的治疗性递送:从设计到治疗应用。

Polymersomes for Therapeutic Delivery of Protein and Nucleic Acid Macromolecules: From Design to Therapeutic Applications.

机构信息

Department of Chemical and Biomolecular Engineering, Clemson University, Clemson, South Carolina 29634, United States.

Department of Bioengineering, Clemson University, Clemson, South Carolina 29634, United States.

出版信息

Biomacromolecules. 2020 Apr 13;21(4):1327-1350. doi: 10.1021/acs.biomac.9b01754. Epub 2020 Mar 2.

Abstract

Macromolecule-based therapeutic agents, particularly proteins, antigens, monoclonal antibodies, transcription factors, nucleic acids, and gene editing enzymes, have the potential to offer cures for previously untreatable diseases. However, they present an enormous delivery challenge due to poor absorption and rapid metabolism in the body. Polymersomes have tremendous potential in delivering these agents to their desired intracellular location due to increased circulation times, decreased macromolecule degradation, and decreased immune responses. In this Review, we highlight the key factors in design, development, and improved performance of these vesicles for macromolecular delivery. The recent progress made toward preclinical application of these vesicles for protein and gene delivery is also covered.

摘要

基于大分子的治疗剂,特别是蛋白质、抗原、单克隆抗体、转录因子、核酸和基因编辑酶,有可能为以前无法治疗的疾病提供治愈方法。然而,由于在体内吸收不良和代谢迅速,它们带来了巨大的给药挑战。聚合物囊泡由于循环时间延长、大分子降解减少和免疫反应减少,在将这些药物递送到所需的细胞内位置方面具有巨大的潜力。在这篇综述中,我们重点介绍了这些囊泡在设计、开发和提高大分子递释性能方面的关键因素。还涵盖了这些囊泡在蛋白质和基因递释方面向临床前应用取得的最新进展。

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