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Engineered IscB-ωRNA System with Improved Base Editing Efficiency for Disease Correction Via Single AAV Delivery in Mice

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

CELL REPORTS(2024)

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关键词
transposon-encoded OMEGA system,RNA-guided nuclease,IscB-ωRNA system,CRISPR ancestry nuclease,cytosine base editing,adenine base editing,gene editing therapy,tyrosinemia,Adeno-associated virus,protein engineering
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