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- W4225497996 abstract "Despite advances in nuclease-based genome editing technologies, correcting human disease-causing genomic inversions remains a challenge. Here, we describe the potential use of a recombinase-based system to correct the 140 kb inversion of the F8 gene frequently found in patients diagnosed with severe Hemophilia A. Employing substrate-linked directed molecular evolution, we develop a coupled heterodimeric recombinase system (RecF8) achieving 30% inversion of the target sequence in human tissue culture cells. Transient RecF8 treatment of endothelial cells, differentiated from patient-derived induced pluripotent stem cells (iPSCs) of a hemophilic donor, results in 12% correction of the inversion and restores Factor VIII mRNA expression. In this work, we present designer-recombinases as an efficient and specific means towards treatment of monogenic diseases caused by large gene inversions." @default.
- W4225497996 created "2022-05-05" @default.
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- W4225497996 date "2022-01-20" @default.
- W4225497996 modified "2023-10-16" @default.
- W4225497996 title "Correction of a Factor VIII genomic inversion with designer-recombinases" @default.
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- W4225497996 doi "https://doi.org/10.1038/s41467-022-28080-7" @default.
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