Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs

Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs

Abstract Hemophilia A (HA) is caused by mutations in coagulation factor VIII (FVIII). Genome editing in conjunction with patient-derived induced pluripotent stem cells (iPSCs) is a promising cell therapy strategy, as it replaces dysfunctional proteins resulting from genetic mutations with normal pro...

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Main Authors: Do-Hun Kim, Sang-Hwi Choi, Jin Jea Sung, Sieun Kim, Hanui Yi, Sanghyun Park, Chan Wook Park, Young Woo Oh, Jungil Lee, Dae-Sung Kim, Jong-Hoon Kim, Chul-Yong Park, Dong-Wook Kim
Format: Article
Language:English
Published: Nature Publishing Group 2025-01-01
Series:Experimental and Molecular Medicine
Online Access:https://doi.org/10.1038/s12276-024-01375-z
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