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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...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7387785/ https://www.ncbi.nlm.nih.gov/pubmed/32711250 http://dx.doi.org/10.1016/j.ebiom.2020.102897 |
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