Biochemical and functional analysis of an adenovirus-based ligand complex for gene transfer

Biochem J. 1994 Apr 1;299 ( Pt 1)(Pt 1):49-58. doi: 10.1042/bj2990049.

Abstract

Ligand-mediated approaches to gene transfer offer an alternative to viral vectors for both in vivo and in vitro applications. Although a significant percentage of the plasmid-based DNA complex is lost to lysosomal degradation following receptor-mediated endocytosis, simultaneous infection with adenovirus has been shown to increase the level of transgene expression [Curiel, Agarwal, Wagner and Cotten (1991) Proc. Natl. Acad. Sci. U.S.A. 88, 8850-8854; Wagner, Zatloukal, Cotten, Kirlappos, Mechtler, Curiel and Birnstiel (1992) Proc. Natl. Acad. Sci. U.S.A. 89, 6099-6103]. In this study we describe an adenovirus-based ligand complex where the plasmid DNA, polycation-ligand conjugate and adenovirus are contained within a single particle structure. At the core of the transfection particle is a replication-defective recombinant adenovirus encoding a cDNA minigene for human placenta alkaline phosphatase that was chemically modified with poly(L-lysine) (Ad-pLys). Electron microscopy of an adenovirus-based ligand complex formed by successively adding plasmid DNA and an asialo-orosomucoid-poly(L-lysine) conjugate to Ad-pLys revealed structures that appeared as intact viral particles coated with a dense biomolecular layer. Adenovirus-based ligand complexes containing either a luciferase or beta-galactosidase reporter plasmid were shown to efficiently deliver the plasmid transgene to cells that express the hepatic asialoglycoprotein receptor. Furthermore, the poly(L-lysine) modification greatly reduced the infectivity potential of the virus without causing a concomitant loss of augmented gene transfer. As an alternative to infectious virions, incomplete products of viral assembly were also considered as a source for endosomalytic activity. However, these defective virions were unable to significantly enhance plasmid transgene delivery.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • 3T3 Cells
  • Adenoviridae / genetics*
  • Adenoviridae / metabolism
  • Alkaline Phosphatase / genetics
  • Alkaline Phosphatase / metabolism
  • Amino Acid Sequence
  • Animals
  • Asialoglycoprotein Receptor
  • Asialoglycoproteins / metabolism
  • Capsid / metabolism
  • Gene Transfer Techniques*
  • Humans
  • Ligands
  • Mice
  • Molecular Sequence Data
  • Orosomucoid / analogs & derivatives
  • Orosomucoid / metabolism
  • Plasmids
  • Polylysine
  • Receptors, Cell Surface / metabolism
  • Transfection
  • Tumor Cells, Cultured

Substances

  • Asialoglycoprotein Receptor
  • Asialoglycoproteins
  • Ligands
  • Orosomucoid
  • Receptors, Cell Surface
  • asialoorosomucoid
  • Polylysine
  • Alkaline Phosphatase