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Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we(More)
We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B. We carried out a phase 1/2 dose-escalation clinical study to extend this approach to humans with severe(More)
  • Katherine A Kay, Bertil Glader High, Frederick A Leonard, Alan Johnson, Ciaran Mcclelland, Erik Scallan +14 others
  • 2002
(517 articles) Gene Therapy (3667 articles) Clinical Trials and Observations Articles on similar topics can be found in the following Blood collections Information about subscriptions and ASH membership may be found online at: digital object identifier (DOIs) and date of initial publication. the indexed by PubMed from initial publication. Citations to(More)
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