Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype

Author:

Sarkar Rita1,Tetreault Renee1,Gao Guangping1,Wang Lili1,Bell Peter1,Chandler Randy1,Wilson James M.1,Kazazian Haig H.1

Affiliation:

1. From the Department of Genetics and Department of Medical Genetics, University of Pennsylvania, Philadelphia.

Abstract

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.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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