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Journal of Virology, June 2006, p. 6192-6194, Vol. 80, No. 12
0022-538X/06/$08.00+0     doi:10.1128/JVI.00526-06
Copyright © 2006, American Society for Microbiology. All Rights Reserved.

High-Level Transgene Expression in Nonhuman Primate Liver with Novel Adeno-Associated Virus Serotypes Containing Self-Complementary Genomes

Guang-Ping Gao,{dagger} You Lu,{dagger} Xun Sun,{dagger} Julie Johnston, Roberto Calcedo, Rebecca Grant, and James M. Wilson*

Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania

Received 14 March 2006/ Accepted 20 March 2006

Adeno-associated virus (AAV) vectors are being considered for in vivo applications of gene therapy in the treatment of a variety of disorders. This study evaluates the biology of second-generation vectors based on the novel serotypes AAV7 and AAV8 and containing self-complementary genomes in the nonhuman primate liver. Stable levels of transgene expression were achieved in cynomolgus macaques and suggest efficiencies at least 2 log higher than what could be achieved with AAV2 vectors using traditional single-stranded genomes. Analysis of DNAs from tissues revealed high levels of vector in the liver that appeared proportional to the relative amounts of transgene expression.

* Corresponding author. Mailing address: Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, 125 South 31st Street, Suite 2000, Philadelphia, PA 19104-3403. Phone: (215) 898-0226. Fax: (215) 898-6588. E-mail: wilsonjm{at}mail.med.upenn.edu.

{dagger} These authors contributed equally to this work.

Journal of Virology, June 2006, p. 6192-6194, Vol. 80, No. 12
0022-538X/06/$08.00+0     doi:10.1128/JVI.00526-06
Copyright © 2006, American Society for Microbiology. All Rights Reserved.



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