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Hematology

Vectors encoding the heavy and light chains of FVIII correct hemophilia A

Published in Proteomics Weekly, January 19th, 2004

Vectors encoding the heavy and light chains of FVIII correct hemophilia A.

According to recent research published in the journal Blood, "using separate adeno-associated viral 2 (AAV2) vectors to deliver the heavy and light chains of factor VIII (FVIII) we have overcome the packaging limitations of AAV, achieving phenotypic correction of hemophilia A in mice. AAV vectors were constructed that use a liver-specific promoter and the cDNA sequences of either the human or canine heavy and light chains of FVIII. After intraportal vein injection of these vectors in hemophilia-A mice, therapeutic to superphysiologic levels of active FVIII were achieved in plasma in a...

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