Article

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

2004-10-13

Abstract excerpt

Abstract In earlier work, we showed that adeno-associated virus–mediated delivery of a Factor IX gene to skeletal muscle by direct intramuscular injection resulted in therapeutic levels of circulating Factor IX in mice. However, achievement of target doses in humans proved impractical because of the large number of injections required. We used a novel intravascular delivery technique to achieve successful transduc...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: da928d5d-1758-5be3-81d0-9789409fb41b
DOI: 10.1182/blood-2004-07-2908

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal modelDOI 10.1182/blood-2004-07-2908Read

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

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

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

Evidence of Multiyear Factor IX Expression by AAV-Mediated Gene Transfer to Skeletal Muscle in an Individual with Severe Hemophilia BSemantic proximity Read

Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia BSemantic proximity Read

Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null MutationSemantic proximity Read

A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in DogsSemantic proximity Read

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

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector.Semantic proximity Read

Evaluation of Vascular Delivery Methodologies to Enhance rAAV6-mediated Gene Transfer to Canine Striated MusculatureSemantic proximity Read

Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?Semantic proximity Read