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

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

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Publicat a:EBioMedicine
Autors principals: 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: Artigo
Idioma:Inglês
Publicat: Elsevier 2020
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7387785/
https://ncbi.nlm.nih.gov/pubmed/32711250
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ebiom.2020.102897
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