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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; Moon, Kyoung-Sik; Lee, Haeshin; Lee, Hyukjin; Yeom, Su Cheong.
Affiliation
  • Han JP; Graduate School of International Agricultural Technology and Institute of Green BioScience and Technology, Seoul National University, Pyeongchang, Gangwon 25354, Korea.
  • Lee Y; College of Pharmacy, Graduate School of Pharmaceutical Sciences, Ewha Womans University, Seodaemun-gu, Seoul 03760, Korea.
  • Lee JH; Graduate School of International Agricultural Technology and Institute of Green BioScience and Technology, Seoul National University, Pyeongchang, Gangwon 25354, Korea.
  • Chung HY; Graduate School of International Agricultural Technology and Institute of Green BioScience and Technology, Seoul National University, Pyeongchang, Gangwon 25354, Korea.
  • Lee GS; Graduate School of International Agricultural Technology and Institute of Green BioScience and Technology, Seoul National University, Pyeongchang, Gangwon 25354, Korea.
  • Nam YR; Deartment of Chemistry, Korea Advanced Institute of Science and Technology (KAIST), Yuseong-gu, Daejeon 34141, Korea.
  • Choi M; Department of Advanced Toxicology Research, Korea Institute of Toxicology, Yuseong-gu, Daejeon 34114, Korea.
  • Moon KS; Department of Advanced Toxicology Research, Korea Institute of Toxicology, Yuseong-gu, Daejeon 34114, Korea.
  • Lee H; Deartment of Chemistry, Korea Advanced Institute of Science and Technology (KAIST), Yuseong-gu, Daejeon 34141, Korea.
  • Lee H; College of Pharmacy, Graduate School of Pharmaceutical Sciences, Ewha Womans University, Seodaemun-gu, Seoul 03760, Korea.
  • Yeom SC; Graduate School of International Agricultural Technology and Institute of Green BioScience and Technology, Seoul National University, Pyeongchang, Gangwon 25354, Korea.
Mol Ther Nucleic Acids ; 34: 102050, 2023 Dec 12.
Article in En | MEDLINE | ID: mdl-37916225
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2023 Document type: Article Country of publication: United States
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2023 Document type: Article Country of publication: United States