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...

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Hauptverfasser: Ying, Ying (VerfasserIn) , Müller, Oliver J. (VerfasserIn) , Göhringer, Caroline (VerfasserIn) , Leuchs, Barbara (VerfasserIn) , Trepel, M. (VerfasserIn) , Katus, Hugo (VerfasserIn) , Kleinschmidt, Jürgen (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 15 April 2010
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
Beschreibung
Zusammenfassung: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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Beschreibung:Online Resource
ISSN:1476-5462
DOI:10.1038/gt.2010.44