In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice

In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice

Gene therapy and rebalancing therapy have emerged as promising approaches for treating hemophilia A, but there are limitations, such as temporary efficacy due to individual differences. Genome editing for hemophilia has shown long-term therapeutic potential in preclinical trials. However, a cautious...

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Bibliographic Details
Main Authors: Jeong Pil Han, Yeji Lee, Jeong Hyeon Lee, Hye Yoon Chung, Geon Seong Lee, Yu Ri Nam, Myeongjin Choi, Kyoung-Sik Moon, Haeshin Lee, Hyukjin Lee, Su Cheong Yeom
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
adeno-associated virus
CRISPR-Cas9
genome editing
hemophilia a
lipid nanoparticle
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123002688

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http://www.sciencedirect.com/science/article/pii/S2162253123002688

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