In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector

• Main Authors: Jeong Hyeon Lee (Author), Jeong Pil Han (Author), Dong Woo Song (Author), Geon Seong Lee (Author), Beom Seok Choi (Author), MinJeong Kim (Author), Yeji Lee (Author), Seokjoong Kim (Author), Hyukjin Lee (Author), Su Cheong Yeom (Author)
• Format: Book
• Published: Elsevier, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Recent therapeutic strategies for hemophilia include long-term therapeutic gene expression using adeno-associated virus (AAV) and rebalancing therapy via the downregulation of anticoagulant pathways. However, these approaches have limitations in immune responses or insufficiency to control acute ble...