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

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

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...

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Bibliographic Details
Main Authors: Jeong Hyeon Lee, Jeong Pil Han, Dong Woo Song, Geon Seong Lee, Beom Seok Choi, MinJeong Kim, Yeji Lee, Seokjoong Kim, Hyukjin Lee, Su Cheong Yeom
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
antithrombin
CRISPR-Cas9
hemophilia B
in vivo knockin
lipid nanoparticle
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123000677

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