Hemophilia Therapy
Endothelial-targeted Sleeping Beauty transposon corrected hemophilia A
Published in Blood Weekly, July 20th, 2006
According to a study from the United States, "Hemophilia A, deficiency of coagulation factor VIII (FVIII), is an attractive candidate for gene therapy as expression of modest amounts of FVIII can provide therapeutic benefit. Most gene transfer approaches for hemophilia have focused on the liver, as this is the major source of endogenous FVIII; however, increasing evidence suggests that endothelial cells are capable of synthesis and release of FVIII. Here the Sleeping Beauty (SB) transposon is employed to target long-term expression of...
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Source: Blood Weekly (2006-07-20)
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