Hematology

New data illuminates research in hematology

Published in Gene Therapy Weekly, June 23rd, 2005

Data on hematology are outlined in reports from the United States and Belgium.

Study 1: Factor IX variants extend the potential of hemophilia B gene therapy

"Intramuscular injection of adeno-associated viral (AAV) vector to skeletal muscle of humans with hemophilia B is safe, but higher doses are required to achieve therapeutic factor IX (F.IX) levels. The efficacy of this approach is hampered by the retention of F.IX in muscle extracellular spaces and by the limiting capacity of muscle to synthesize fully active F.IX at high expression rates," investigators in the United States report.

"To overcome these limitations, we...

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Source: Gene Therapy Weekly (2005-06-23)

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