In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice-Reference-Cited by-同舟云学术

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

Published:2023-12 Issue: Volume:34 Page:102050
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Han Jeong Pil,Lee Yeji,Lee Jeong Hyeon,Chung Hye Yoon,Lee Geon Seong,Nam Yu Ri,Choi Myeongjin,Moon Kyoung-Sik,Lee Haeshin,Lee Hyukjin^ORCID,Yeom Su Cheong^ORCID

Funder

Ewha Womans University

Ministry of Food and Drug Safety

Ministry of Science, ICT and Future Planning

Korea Institute of Toxicology

National Research Foundation of Korea

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

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4. The impact of extended half-life factor concentrates on prophylaxis for severe hemophilia in the United States;Malec;Am. J. Hematol.,2020

5. Recombinant factor VIII products and inhibitor development in previously untreated patients with severe haemophilia A: Combined analysis of three studies;Volkers;Haemophilia,2019

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