Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library
Selection of targeted vectors from virus display peptide libraries is a versatile and efficient approach to improve vector specificity and efficiency. This strategy has been used to target various cell types in vitro. Here, we report the screening of an adeno-associated virus type 2 (AAV2) display p...
Gespeichert in:
| Hauptverfasser: | , , , , , , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
15 April 2010
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| In: |
Gene therapy
Year: 2010, Jahrgang: 17, Heft: 8, Pages: 980-990 |
| ISSN: | 1476-5462 |
| DOI: | 10.1038/gt.2010.44 |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/gt.2010.44 Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/gt201044 
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| Verfasserangaben: | Y. Ying, O.J. Müller, C. Goehringer, B. Leuchs, M. Trepel, H.A. Katus and J.A. Kleinschmidt |
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| 520 | |a Selection of targeted vectors from virus display peptide libraries is a versatile and efficient approach to improve vector specificity and efficiency. This strategy has been used to target various cell types in vitro. Here, we report the screening of an adeno-associated virus type 2 (AAV2) display peptide library in vivo to select vectors specifically homing to heart tissue after systemic application in mice. Selected library clones indicated superior specificity of gene transfer compared with wild-type AAV2, AAV9 and a heparin binding-deficient AAV2 mutant. Such targeted vectors were able to reconstitute expression of δ-sarcoglycan in the heart of adult δ-sarcoglycan knockout mice after systemic gene transfer in vivo, attesting to the therapeutic potential of this approach. | ||
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