Hematology
Adeno-associated virus extends gene transfection in hemophilia models
Published in Blood Weekly, April 3rd, 2003
"The goal of our work has been to establish an experimental basis for gene transfer as a method of treating hemophilia, an inherited bleeding disorder that results from the absence of functional factor VIII or factor IX," said K. High and colleagues, University of Pennsylvania, Children's Hospital of Philadelphia.
"Using an adeno-associated viral vector derived from AAV serotype 2, we have shown in mice and in hemophilic dogs that we can achieve long-term expression (>3 years) of clotting factor at levels that would result in an improvement of clinical symptoms of the disease,"...
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Source: Blood Weekly (2003-04-03)
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