An effective vaginal gel to deliver CRISPR/Cas9 system encapsulated in poly (β-amino ester) nanoparticles for vaginal gene therapy

• Published in: EBioMedicine Vol. 58; p. 102897
• Main Authors: Niu, Gang, Jin, Zhuang, Zhang, Chong, He, Dan, Gao, Xueqin, Zou, Chenming, Zhang, Wei, Ding, Jiahui, Das, Bhudev C., Severinov, Konstantin, Hitzeroth, Inga Isabel, Debata, Priya Ranjan, Ma, Xin, Tian, Xun, Gao, Qinglei, Wu, Jun, You, Zeshan, Tian, Rui, Cui, Zifeng, Fan, Weiwen, Xie, Weiling, Huang, Zhaoyue, Cao, Chen, Xu, Wei, Xie, Hongxian, Xu, Hongyan, Tang, Xiongzhi, Wang, Yan, Yu, Zhiying, Han, Hui, Tan, Songwei, Chen, Shuqin, Hu, Zheng
• Format: Journal Article
• Language: English
• Published: Netherlands Elsevier B.V 01.08.2020; Elsevier
• Subjects: Administration, Intravaginal; Advanced Basic Science; Animals; Bentonite - chemistry; Cells, Cultured; Cervix Uteri - chemistry; Cervix Uteri - cytology; CRISPR-Cas Systems; CRISPR/Cas9; Endogenous Retroviruses - drug effects; Endogenous Retroviruses - genetics; Female; Gene Dosage - drug effects; Gene Editing; Gene therapy; Genetic Therapy; Internal Medicine; Local delivery; Mice; Models, Animal; Nanoparticles; Plasmids - administration & dosage; Plasmids - genetics; Poly (β-amino ester); Polymers - chemistry; Research paper; RNA, Guide, CRISPR-Cas Systems - administration & dosage; Silicates - chemistry; Swine; Vaginal Creams, Foams, and Jellies; Vaginal gel; Local delivery; Poly (β-amino ester); Vaginal gel; CRISPR/Cas9; Gene therapy

Gene therapy has held promises for treating specific genetic diseases. However, the key to clinical application depends on effective gene delivery. Using a large animal model, we developed two pharmaceutical formulations for gene delivery in the pigs’ vagina, which were made up of poly (β-amino este...