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Blood. 2012 Mar 29;119(13):2973-4. doi: 10.1182/blood-2012-02-409086.

Gene therapy, an ongoing revolution.

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  • Université Pierre et Marie Curie.

Abstract

In this issue of Blood, Buchlis and colleagues describe the long-term persistence (up to 10 years) of factor IX (FIX) expression in adeno-associated virus serotype 2 (AAV-2)–injected muscles of a patient with hemophilia B.

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PMID:
22461470
[PubMed - indexed for MEDLINE]
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