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...

Full description

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

Internet

http://www.sciencedirect.com/science/article/pii/S2162253123000677

Similar Items