Zhang Jiayuan, Yang Xinyu, Chang Zhichao, Zhu Wenwei, Ma Yuhua, He Haisheng
Key Laboratory of Smart Drug Delivery of Ministry of Education, School of Pharmacy, Fudan University, Shanghai 201203, China.
Key Laboratory for Tibet Plateau Phytochemistry of Qinghai Province, School of Pharmacy, Qinghai Minzu University, Xining 810007, China.
Asian J Pharm Sci. 2025 Feb;20(1):101015. doi: 10.1016/j.ajps.2025.101015. Epub 2025 Jan 4.
The recent commercialization of gene products has sparked significant interest in gene therapy, necessitating efficient and precise gene delivery via various vectors. Currently, viral vectors and lipid-based nanocarriers are the predominant choices and have been extensively investigated and reviewed. Beyond these vectors, polymeric nanocarriers also hold the promise in therapeutic gene delivery owing to their versatile functionalities, such as improving the stability, cellar uptake and endosomal escape of nucleic acid drugs, along with precise delivery to targeted tissues. This review presents a brief overview of the status quo of the emerging polymeric nanocarriers for therapeutic gene delivery, focusing on key cationic polymers, nanocarrier types, and preparation methods. It also highlights targeted diseases, strategies to improve delivery efficiency, and potential future directions in this research area. The review is hoped to inspire the development, optimization, and clinical translation of highly efficient polymeric nanocarriers for therapeutic gene delivery.
基因产品最近的商业化引发了人们对基因治疗的浓厚兴趣，这就需要通过各种载体进行高效且精确的基因递送。目前，病毒载体和基于脂质的纳米载体是主要选择，并且已经得到了广泛的研究和综述。除了这些载体之外，聚合物纳米载体因其具有多种功能，如提高核酸药物的稳定性、细胞摄取和内体逃逸能力，以及精确递送至靶向组织，在治疗性基因递送方面也具有前景。本综述简要概述了用于治疗性基因递送的新型聚合物纳米载体的现状，重点介绍了关键阳离子聚合物、纳米载体类型和制备方法。它还强调了靶向疾病、提高递送效率的策略以及该研究领域潜在的未来方向。希望这篇综述能激发用于治疗性基因递送的高效聚合物纳米载体的开发、优化和临床转化。
