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

Jeong Pil Han, Yeji Lee, Jeong Hyeon Lee, Hye Yoon Chung, Geon Seong Lee, Yu Ri Nam, Myeongjin Choi, Kyoung Sik Moon, Haeshin Lee, Hyukjin Lee, Su Cheong Yeom

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology