Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library

Gene Ther. 2010 Aug;17(8):980-90. doi: 10.1038/gt.2010.44. Epub 2010 Apr 15.

Abstract

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line
  • Dependovirus / genetics*
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Mice
  • Myocardium / metabolism*
  • Myocytes, Cardiac / metabolism
  • Myocytes, Cardiac / virology
  • Peptide Library
  • Rats
  • Sarcoglycans / genetics*
  • Transduction, Genetic

Substances

  • Peptide Library
  • Sarcoglycans