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Blood, 15 February 2004, Vol. 103, No. 4, pp. 1253-1260.
Prepublished online as a Blood First Edition Paper on October 9, 2003; DOI 10.1182/blood-2003-08-2954.
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GENE THERAPY
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype
Rita Sarkar,
Renee Tetreault,
Guangping Gao,
Lili Wang,
Peter Bell,
Randy Chandler,
James M. Wilson, and
Haig H. Kazazian, Jr
From the Department of Genetics and Department of Medical Genetics, University of Pennsylvania, Philadelphia.
Despite the popularity of adeno-associated virus 2 (AAV2) as a vehicle for gene transfer, its efficacy for liver-directed gene therapy in hemophilia A or B has been suboptimal. Here we evaluated AAV serotypes 2, 5, 7, and 8 in gene therapy of factor VIII (FVIII) deficiency in a hemophilia A mouse model and found that AAV8 was superior to the other 3 serotypes. We expressed canine B domain-deleted FVIII cDNA either in a single vector or in 2 separate AAV vectors containing the heavy- and light-chain cDNAs. We also evaluated AAV8 against AAV2 in intraportal and tail vein injections. AAV8 gave 100% correction of plasma FVIII activity irrespective of the vector type or route of administration.
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