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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Main Authors: Ying, Ying (Author) , Müller, Oliver J. (Author) , Göhringer, Caroline (Author) , Leuchs, Barbara (Author) , Trepel, M. (Author) , Katus, Hugo (Author) , Kleinschmidt, Jürgen (Author)
Format: Article (Journal)
Language:English
Published: 15 April 2010
In: Gene therapy
Year: 2010, Volume: 17, Issue: 8, Pages: 980-990
ISSN:1476-5462
DOI:10.1038/gt.2010.44
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/gt.2010.44
Verlag, lizenzpflichtig, Volltext: https://www.nature.com/articles/gt201044
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Author Notes:Y. Ying, O.J. Müller, C. Goehringer, B. Leuchs, M. Trepel, H.A. Katus and J.A. Kleinschmidt
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Summary: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.
Item Description:Gesehen am 03.07.2024
Physical Description:Online Resource
ISSN:1476-5462
DOI:10.1038/gt.2010.44