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Engineered IscB-ωRNA system with improved base editing efficiency for disease correction via single AAV delivery in mice

Summary: IscBs, as hypercompact ancestry proteins of Cas9 nuclease, are suitable for in vivo gene editing via single adeno-associated virus (AAV) delivery. Due to the low activity of natural IscBs in eukaryotic cells, recent studies have been focusing on improving OgeuIscB’s gene editing efficiency...

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Bibliografiske detaljer
Principais autores: Ruochen Guo, Xiaozhi Sun, Feizuo Wang, Dingyi Han, Qiaoxia Yang, Hua Gao, Zhifang Li, Zhuang Shao, Jinqi Shi, Rongrong Yang, Xiaona Huo, Junda Yan, Guoling Li, Qingquan Xiao, Yuanhua Liu, Senfeng Zhang, Xinyu Liu, Yingsi Zhou, Leyun Wang, Chunyi Hu, Chunlong Xu
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2024-11-01
Serier:Cell Reports
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Online adgang:http://www.sciencedirect.com/science/article/pii/S221112472401324X
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