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Article

Robust ZFN-mediated genome editing in adult hemophilic mice

2 Oct 2013

Abstract excerpt

Monogenic diseases, including hemophilia, represent ideal targets for genome-editing approaches aimed at correcting a defective gene. Here we report that systemic adeno-associated virus (AAV) vector delivery of zinc finger nucleases (ZFNs) and corrective donor template to the predominantly quiescent livers of adult mice enables production of high levels of human factor IX in a murine model of hemophilia B....

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PMID: 24085764
DOI: 10.1182/blood-2013-04-497354

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