Beta-amino ester polymers facilitate in vivo DNA transfection and adjuvant plasmid DNA immunization

Mol Ther. 2005 Jul;12(1):164-70. doi: 10.1016/j.ymthe.2005.01.021.

Abstract

Increased in vivo expression of intramuscularly delivered plasmid DNA will be essential for clinical success in gene therapy and plasmid DNA vaccination. We screened polymers from a library of beta-amino esters for their ability to augment transgene expression as measured by beta-galactosidase activity and cellular immune responses. Among the candidates identified in this screen, poly[(1,6-di(acryloxyethoxy)hexane)-co-(4-aminobutanol)] enhanced plasmid DNA transgene expression by sevenfold (P=0.0001) and its immunogenicity by 70% (P=0.03). We found that polymers with moderately hydrophobic backbones and terminal alcohol groups facilitated transfection most effectively in vivo. We also observed a log-linear correlation (R2=0.93) between peak cellular immune responses and transgene activity in all evaluated polymer-plasmid DNA formulations, clarifying the relationship between immunogenicity and the quantity of expressed antigen.

Publication types

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

MeSH terms

  • Animals
  • Antigens / immunology
  • Immunity, Cellular
  • Mice
  • Muscles / immunology
  • Plasmids* / immunology
  • Polymers
  • Time Factors
  • Transfection*
  • Vaccines, DNA* / immunology
  • beta-Galactosidase / genetics
  • beta-Galactosidase / metabolism

Substances

  • Antigens
  • CRL 1005
  • Polymers
  • Vaccines, DNA
  • beta-Galactosidase