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Hemophilia gene therapy comes of age

8 Dec 2017

Abstract excerpt

genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX activity in 2011, and...

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PMID: 29222308
DOI: 10.1182/asheducation-2017.1.587

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