Abstract
Gene therapy for the X-linked bleeding disorder hemophilia B may be limited by immune responses to the factor IX (F.IX) gene product. Hepatic adeno-associated virus (AAV) gene transfer can induce immune tolerance to F.IX (
JCI
111
:1347
PNAS
103
:4592
Disclosures: RW Herzog has received royalties from Genzyme Corp. for AAV-F.IX technology.
Author notes
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Corresponding author
2006, The American Society of Hematology
2006
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