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Article

Therapeutic Levels of Factor IX Expression Using a Muscle-Specific Promoter and Adeno-Associated Virus Serotype 1 Vector

1 Aug 2004

Abstract excerpt

Extensive studies in animal models of the X-linked bleeding disorder hemophilia B (deficiency in functional coagulation factor IX, F.IX) have shown that muscle-directed adeno-associated (AAV)-mediated F.IX gene transfer can be used to treat this disease. However, large vector doses of AAV-2 vector are required for therapeutic levels of expression, and the number of vector doses that can be injected per...

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PMID: 15319035
DOI: 10.1089/1043034041648453

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Therapeutic Levels of Factor IX Expression Using a Muscle-Specific Promoter and Adeno-Associated Virus Serotype 1 VectorPMID 15319035Read ↗

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1Semantic proximity Read ↗

Factor IX variants improve gene therapy efficacy for hemophilia BSemantic proximity Read ↗

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia BSemantic proximity Read ↗

Role of Vector in Activation of T Cell Subsets in Immune Responses against the Secreted Transgene Product Factor IXSemantic proximity Read ↗

Muscle as a Target for Supplementary Factor IX Gene TransferSemantic proximity Read ↗

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in DogsSemantic proximity Read ↗

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal modelSemantic proximity Read ↗

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.Semantic proximity Read ↗

Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia BSemantic proximity Read ↗

Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.Semantic proximity Read ↗

Several Log Increase in Therapeutic Transgene Delivery by Distinct Adeno-Associated Viral Serotype VectorsSemantic proximity Read ↗

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vectorSemantic proximity Read ↗

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