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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Detalhes bibliográficos
Publicado no:EBioMedicine
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
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2020
Assuntos:
Research paper
Acesso em linha: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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