Hemophilia Therapy

Adenoviral gene therapy mediates correction of defect in canine hemophilia A model

Published in Gene Therapy Weekly, July 20th, 2006

Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model.

According to a study from the United States, "Adenoviral vector-mediated gene therapy might have potential for long-term correction of the monogenic disease hemophilia A. In this study, we tested the efficacy of administering a helper-dependent adenoviral vector (HDV) designed for maximal liver-restricted canine factor VIII (cFVIII) expression on three out-bred hemophilia A dogs."

"Three FVIII-deficient animals from the University of North Carolina colony were injected with 1 x 10¹² (Dog A), and 3 x...

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Source: Gene Therapy Weekly (2006-07-20)

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