Cell targeting peptide conjugation to siRNA polyplexes for effective gene silencing in cardiomyocytes

Mol Pharm. 2012 May 7;9(5):1302-9. doi: 10.1021/mp200589z. Epub 2012 Apr 19.

Abstract

To deliver siRNA specifically to cardiomyocytes with a high transfection efficiency, primary cardiomyocyte-targeting (PCM) and/or cell-penetrating (Tat) peptides were incorporated into the siRNA. With the addition of plasmid DNA, these peptide-conjugated siRNAs were able to form compact and stable nanosized polyplex particles with bioreducible poly(CBA-DAH). The peptide-modified siRNA polyplexes enhanced the cellular uptake and the gene-silencing capacity of the siRNA in cardiomyocytes without significant immunogenicity or cytotoxicity. These findings demonstrate that the cell-targeting peptide and/or cell-penetrating peptide conjugation of siRNA may be a potentially important strategy for cell-specific gene therapy in gene-mediated disease states.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Cell Line
  • Cell-Penetrating Peptides / chemistry*
  • Gene Silencing*
  • Genetic Therapy
  • Humans
  • Microscopy, Electron, Scanning
  • Myocytes, Cardiac / metabolism*
  • Polymers / administration & dosage*
  • Polymers / chemistry*
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / chemistry*
  • Transfection / methods*

Substances

  • Cell-Penetrating Peptides
  • Polymers
  • RNA, Small Interfering