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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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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
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