Article

Correction of a Factor VIII genomic inversion with designer-recombinases

2020-11-02

Abstract excerpt

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 a 140 kb genomic inversion of the F8 gene, which is frequently found in patients diagnosed with severe Hemophilia A. Employing substrate-linked directed molecular evolution, we developed a fused heterodi...

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Literature Corpus work: 53bf65f0-4e77-5932-b9b5-8efd2869f78d
DOI: 10.1101/2020.11.02.328013

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