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

• Главные авторы: Jeong Pil Han (Автор), Yeji Lee (Автор), Jeong Hyeon Lee (Автор), Hye Yoon Chung (Автор), Geon Seong Lee (Автор), Yu Ri Nam (Автор), Myeongjin Choi (Автор), Kyoung-Sik Moon (Автор), Haeshin Lee (Автор), Hyukjin Lee (Автор), Su Cheong Yeom (Автор)
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• Опубликовано: Elsevier, 2023-12-01T00:00:00Z.
• Предметы: article
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Gene therapy and rebalancing therapy have emerged as promising approaches for treating hemophilia A, but there are limitations, such as temporary efficacy due to individual differences. Genome editing for hemophilia has shown long-term therapeutic potential in preclinical trials. However, a cautious...