Virus-mimetic polyplex particles for systemic and inflammation-specific targeted delivery of large genetic contents

S. Kang, K. Lu, J. Leelawattanachai, X. Hu, S. Park, T. Park, I. M. Min, M. M. Jin

Research output: Contribution to journalArticlepeer-review

11 Scopus citations


Systemic and target-specific delivery of large genetic contents has been difficult to achieve. Although viruses effortlessly deliver kilobase-long genome into cells, its clinical use has been hindered by serious safety concerns and the mismatch between native tropisms and desired targets. Nonviral vectors, in contrast, are limited by low gene transfer efficiency and inherent cytotoxicity. Here we devised virus-mimetic polyplex particles (VMPs) based on electrostatic self-Assembly among polyanionic peptide (PAP), cationic polymer polyethyleneimine (PEI) and nucleic acids. We fused PAP to the engineered ligand-binding domain of integrin L β 2 to target intercellular adhesion molecule-1 (ICAM-1), an inducible marker of inflammation. Fully assembled VMPs packaged large genetic contents, bound specifically to target molecules, elicited receptor-mediated endocytosis and escaped endosomal pathway, resembling intracellular delivery processes of viruses. Unlike conventional PEI-mediated transfection, molecular interaction-dependent gene delivery of VMPs was unaffected by the presence of serum and achieved higher efficiency without toxicity. By targeting overexpressed ICAM-1, VMPs delivered genes specifically to inflamed endothelial cells and macrophages both in vitro and in vivo. Simplicity and versatility of the platform and inflammation-specific delivery may open up opportunities for multifaceted gene therapy that can be translated into the clinic and treat a broad range of debilitating immune and inflammatory diseases. Copy; 2013 Macmillan Publishers Limited.

Original languageEnglish (US)
Pages (from-to)1042-1052
Number of pages11
JournalGene Therapy
Issue number11
StatePublished - Nov 2013


  • ICAM-1
  • inflammation
  • integrin LFA-1
  • targeted gene delivery
  • virus-mimetic polyplex particles

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics


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