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 |
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Format: | Article |
Language: | English |
Published: |
Nature Publishing Group
2025-01-01
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Series: | Experimental and Molecular Medicine |
Online Access: | https://doi.org/10.1038/s12276-024-01375-z |
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