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

6 Jan 2025

Abstract excerpt

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 proteins. However, the low expression level and short half-life of FVIII still remain significant limiting factors in the...

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CAR-T cell therapy research
CRISPR and Genetic Engineering
Pluripotent Stem Cells Research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39762408
DOI: 10.1038/s12276-024-01375-z

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