Hemophilia A

Research from B.W. Peng and co-researchers yields new findings on hemophilia a

Published in Gene Therapy Weekly, October 23rd, 2008

"Formation of inhibitory antibodies is a common problem encountered in clinical treatment for hemophilia. Human factor VIII(hFVIII) plasmid genetherapy in hemophilia A mice also leads to strong humoral responses," scientists writing in the journal Blood report.

"We demonstrate that short-term therapy with an anti-ICOS monoclonal antibody to transiently block the inducible costimulator/inducible costimulator ligand (ICOS/ICOSL) signaling pathway led to sustained tolerance to hFVIII in hFVIII plasmid-treated hemophilia A mice and allowed persistent, high-level FVIII functional activity (100%-300% of normal). Anti-ICOS treatment resulted in depletion of...

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Source: Gene Therapy Weekly (2008-10-23)

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