Journal
GENE THERAPY
Volume 17, Issue 8, Pages 980-990Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/gt.2010.44
Keywords
AAV; vector targeting; heart
Categories
Funding
- Deutsche Forschungsgemeinschaft [KL 516/8-2, TR-448/5-3, Mu 1654/3-2]
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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 delta-sarcoglycan in the heart of adult delta-sarcoglycan knockout mice after systemic gene transfer in vivo, attesting to the therapeutic potential of this approach. Gene Therapy (2010) 17, 980-990; doi: 10.1038/gt.2010.44; published online 15 April 2010
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