Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung

Proc Natl Acad Sci U S A. 2005 Apr 19;102(16):5679-84. doi: 10.1073/pnas.0502067102. Epub 2005 Apr 11.

Abstract

High-molecular-mass polyethylenimines (PEIs) are widely used vectors for nucleic acid delivery. We found that removal of the residual N-acyl moieties from commercial linear 25-kDa PEI enhanced its plasmid DNA delivery efficiency 21 times in vitro, as well as 10,000 times in mice with a concomitant 1,500-fold enhancement in lung specificity. Several additional linear PEIs were synthesized by acid-catalyzed hydrolysis of poly(2-ethyl-2-oxazoline), yielding the pure polycations. PEI87 and PEI217 exhibited the highest efficiency in vitro: 115-fold and 6-fold above those of the commercial and deacylated PEI25s, respectively; moreover, PEI87 delivered DNA to mouse lung as efficiently as the pure PEI25 but at a lower concentration and with a 200-fold lung specificity. These improvements stem from an increase in the number of protonatable nitrogens, which presumably results in a tighter condensation of plasmid DNA and a better endosomal escape of the PEI/DNA complexes. As a validation of the potential of such linear, fully deacylated PEIs in gene therapy for lung diseases, systemic delivery in mice of the complexes of a short interfering RNA (siRNA) against a model gene, firefly luciferase, and PEI25 or PEI87 afforded a 77% and 93% suppression of the gene expression in the lungs, respectively. Furthermore, a polyplex of a siRNA against the influenza viral nucleocapsid protein gene and PEI87 resulted in a 94% drop of virus titers in the lungs of influenza-infected animals.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Acylation
  • Animals
  • Cell Line, Tumor
  • Gene Expression Regulation
  • Gene Transfer Techniques*
  • Humans
  • Influenza, Human / drug therapy
  • Influenza, Human / genetics
  • Lung / metabolism*
  • Mice
  • Orthomyxoviridae / drug effects
  • Orthomyxoviridae / metabolism
  • Polyethyleneimine / chemistry*
  • Polyethyleneimine / metabolism*
  • Polyethyleneimine / therapeutic use
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism
  • RNA, Small Interfering / pharmacology
  • RNA, Small Interfering / therapeutic use

Substances

  • RNA, Small Interfering
  • Polyethyleneimine