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Article

Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B

1 Jan 2011

Abstract excerpt

Intramuscular (IM) administration of an adeno-associated viral (AAV) vector represents a simple and safe method of gene transfer for treatment of the X-linked bleeding disorder hemophilia B (factor IX, F.IX, deficiency). However, the approach is hampered by an increased risk of immune responses against F.IX. Previously, we demonstrated that the drug cocktail of immune suppressants rapamycin, IL-10, and a specific...

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Identifiers

PMID: 22279442
DOI: 10.3389/fmicb.2011.00244

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