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Article

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

9 Dec 2017

Abstract excerpt

BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia B, the large size of the factor VIII coding region has precluded improved outcomes with gene therapy in patients with hemophilia A. METHODS: We infused a single intravenous dose of a...

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PMID: 29224506
DOI: 10.1056/nejmoa1708483

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