PB0165 Gene Correction of Human Hemophilia B Mutation by PAM-Flexible Cas9-Mediated Base-Editing Approach

T. Hiramoto, Y. Kashiwakura, M. Hayakawa,N. Baatartsogt, N. Kamoshita,T. Abe, H. Inaba, H. Nishimasu,H. Uosaki,Y. Hanazono, O. Nureki,T. Ohmori

Research and Practice in Thrombosis and Haemostasis(2023)

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pb0165 gene correction,hemophilia,mutation,pam-flexible,base-editing
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