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Jing Zhang, Zhonglei He, Yinghao Li, Yu Shen, Guanfu Wu, Laura Power, Rijian Song, Ming Zeng, Xianqing Wang, Irene Lara Sáez, Sigen A, Qian Xu, James F. Curtin, Ziyi Yu, Wenxin Wang, Enhanced gene transfection efficacy and safety through granular hydrogel mediated gene delivery process, Acta Biomaterialia, Volume 166, 2023, Pages 85-94.


Although gene therapy has made great achievements in both laboratory research and clinical translation, there are still challenges such as limited control of drug pharmacokinetics, acute toxicity, poor tissue retention, insufficient efficacy, and inconsistent clinical translation. Herein, a gene therapy gel is formulated by directly redispersing polyplex nanoparticles into granular hydrogels without any gelation pre-treatment, which provides great convenience for storage, dosing and administration. In vitro studies have shown that use of granular hydrogels can regulate the gene drug release, reduce dose dependent toxicity and help improve transfection efficacy. Moreover, the developed gene therapy gel is easy to operate and can be directly used in vitro to evaluate its synergistic efficacy with various gene delivery systems. As such, it represents a major advance over many conventional excipient-based formulations, and new regulatory strategies for gene therapy may be inspired by it.


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Creative Commons Attribution-Share Alike 4.0 International License
This work is licensed under a Creative Commons Attribution-Share Alike 4.0 International License.