Hemophilia

Gene therapy induces tolerance to factor VIII inhibitors

Published in Blood Weekly, August 18th, 2005

Researchers have demonstrated induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins.

According to a study from the United States, "Up to 30% of patients with hemophilia A given therapeutic factor VIII (fVIII) can make inhibitory antibodies, the majority of which are reactive with its C2 and A2 domains. We have previously demonstrated that anti-gen-specific tolerance to several antigens can be induced by lipopolysaccharide (LPS)-activated B-cell blasts transduced with immunoglobulin (IgG)-antigen fusion constructs."

"To apply this system to hemophilia A...

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Source: Blood Weekly (2005-08-18)

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