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Article

Muscle as a Target for Supplementary Factor IX Gene Transfer

26 Jun 2007

Abstract excerpt

Immune responses to the factor IX (F.IX) transgene product are a concern in gene therapy for the X-linked bleeding disorder hemophilia B. The risk for such responses is determined by several factors, including the vector, target tissue, and others. Previously, we have demonstrated that hepatic gene transfer with adeno-associated viral (AAV) vectors can induce F.IX-specific immune tolerance. Muscle-derived F.IX...

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Identifiers

PMID: 17594244
DOI: 10.1089/hum.2007.042

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Muscle as a Target for Supplementary Factor IX Gene TransferPMID 17594244Read ↗

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