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Journal of Virology, January 2002, p. 791-801, Vol. 76, No. 2
0022-538X/01/$04.00+0     DOI: 10.1128/JVI.76.2.791-801.2002
Copyright © 2002, American Society for Microbiology. All Rights Reserved.

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

Joseph E. Rabinowitz,1 Fabienne Rolling,2 Chengwen Li,1 Hervè Conrath,2 Weidong Xiao,3 Xiao Xiao,4 and R. Jude Samulski1,5*

Gene Therapy Center,1 Laboratoire de Thérapie Génique, CHU Hotel-DIEU, 44035 Nantes Cedex 01, France,2 Division of Hematology, Department of Pediatrics, University of Pennsylvania School of Medicine and Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania,3 Department of Microbiology, University of Pittsburgh, Pittsburgh, Pennsylvania,4 Department of Pharmacology, University of North Carolina, Chapel Hill, North Carolina5

Received 7 August 2001/ Accepted 9 October 2001

The serotypes of adeno-associated virus (AAV) have the potential to become important resources for clinical gene therapy. In an effort to compare the role of serotype-specific virion shells on vector transduction, we cloned each of the serotype capsid coding domains into a common vector backbone containing AAV type 2 replication genes. This strategy allowed the packaging of AAV2 inverted terminal repeat vectors into each serotype-specific virions. Each of these helper plasmids (pXR1 through pXR5) efficiently replicated the transgene DNA and expressed helper proteins at nearly equivalent levels. In this study, we observed a correlation between the amount of transgene replication and packaging efficiency. The physical titer of these hybrid vectors ranged between 1.3 x 1011 and 9.8 x 1012/ml (types 1 and 2, respectively). Of the five serotype vectors, only types 2 and 3 were efficiently purified by heparin-Sepharose column chromatography, illustrating the high degree of similarity between these virions. We analyzed vector transduction in reference and mutant Chinese hamster ovary cells deficient in heparan sulfate proteoglycan and saw a correlation between transduction and heparan sulfate binding data. In this analysis, types 1 and 5 were most consistent in transduction efficiency across all cell lines tested. In vivo each serotype was ranked after comparison of transgene levels by using different routes of injection and strains of rodents. Overall, in this analysis, type 1 was superior for efficient transduction of liver and muscle, followed in order by types 5, 3, 2, and 4. Surprisingly, this order changed when vector was introduced into rat retina. Types 5 and 4 were most efficient, followed by type 1. These data established a hierarchy for efficient serotype-specific vector transduction depending on the target tissue. These data also strongly support the need for extending these analyses to additional animal models and human tissue. The development of these helper plasmids should facilitate direct comparisons of serotypes, as well as begin the standardization of production for further clinical development.


* Corresponding author. Mailing address: Gene Therapy Center, The University of North Carolina at Chapel Hill, 7119 Thurston/Bowles CB7352, Chapel Hill, NC 27599-7352. Phone: (919) 966-0191. Fax: (919) 966-0907. E-mail: rjs{at}med.unc.edu.


Journal of Virology, January 2002, p. 791-801, Vol. 76, No. 2
0022-538X/01/$04.00+0     DOI: 10.1128/JVI.76.2.791-801.2002
Copyright © 2002, American Society for Microbiology. All Rights Reserved.




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