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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Autors principals:	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:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2023-06-01
Col·lecció:	Molecular Therapy: Nucleic Acids
Matèries:	MT: Oligonucleotides: Therapies and Applications antithrombin CRISPR-Cas9 hemophilia B in vivo knockin lipid nanoparticle
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2162253123000677
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