Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer |
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| Authors: | Hildinger M Auricchio A Gao G Wang L Chirmule N Wilson J M |
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| Affiliation: | Institute for Human Gene Therapy, Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. |
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| Abstract: | Vectors based on hybrids consisting of adeno-associated virus types 2 (ITRs and Rep) and 5 (Cap) were evaluated for muscle-directed gene transfer (called AAV2/5). Evaluation in immune-competent mice revealed greater transduction efficacy with AAV2/5 than with AAV2 and no cross-neutralization between AAV2/5 and AAV2. Interestingly, we saw no immunologic evidence of previous exposure to AAV5 capsids in a large population of healthy human subjects. |
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