Published in Gene Therapy Weekly, November 23rd, 2000
"Hemophilia A is an X-linked bleeding disorder caused by lack of functional factor VIII protein, making it a good model disease for gene therapy," stated S. Kobayashi and colleagues from the University of Pennsylvania. "Epidermis is an attractive target tissue for gene therapy because it is accessible and gene products expressed there can enter the systemic circulation.
"Previously, we generated transgenic mice that express human B domain-deleted factor VIII in the suprabasal...
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