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In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice

Han, Jeong Pil; Lee, Yeji; Lee, Jeong Hyeon; Chung, Hye Yoon; Lee, Geon Seong; Nam, Yu Ri; Choi, Myeongjin; et al, MOLECULAR THERAPY-NUCLEIC ACIDS, v.34, 2023-12

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