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

• Main Authors: Jeong Pil Han (Author), Yeji Lee (Author), Jeong Hyeon Lee (Author), Hye Yoon Chung (Author), Geon Seong Lee (Author), Yu Ri Nam (Author), Myeongjin Choi (Author), Kyoung-Sik Moon (Author), Haeshin Lee (Author), Hyukjin Lee (Author), Su Cheong Yeom (Author)
• Format: Book
• Published: Elsevier, 2023-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...