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, Haig H. Kazazian

Research output: Contribution to journalArticlepeer-review

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1253-1260
Number of pages8
JournalBlood
Volume103
Issue number4
DOIs
StatePublished - Feb 15 2004
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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