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

• প্রধান লেখক: Jeong Hyeon Lee (লেখক), Jeong Pil Han (লেখক), Dong Woo Song (লেখক), Geon Seong Lee (লেখক), Beom Seok Choi (লেখক), MinJeong Kim (লেখক), Yeji Lee (লেখক), Seokjoong Kim (লেখক), Hyukjin Lee (লেখক), Su Cheong Yeom (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: Elsevier, 2023-06-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: 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...