Gene therapy, an ongoing revolution
- PMID: 22461470
- DOI: 10.1182/blood-2012-02-409086
Gene therapy, an ongoing revolution
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.
Comment on
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Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23. Blood. 2012. PMID: 22271447 Free PMC article.
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