An autoantibody is modified for use as a delivery system to target the cell nucleus: therapeutic implications

J Autoimmun. 1998 Oct;11(5):539-46. doi: 10.1006/jaut.1998.0212.

Abstract

A murine monoclonal anti-dsDNA antibody was found to penetrate living cells and localize in the nucleus without pathologic effects. A single mutation in VH markedly enhanced cellular penetration. The mutant antibody was produced as recombinant Fab and single chain antibody fragments to investigate its use as a delivery system to target the cell nucleus. Complexes were made containing Fab fragments and alkaline phosphatase conjugated goat antibodies to mouse |gk chains. Fab fragments transported 305 kDa goat antibody-enzyme complexes into the nucleus in COS-7 and CHO cells. A single chain antibody cDNA was constructed by splice overlap extension PCR and expressed in COS-7 cells. Binding of the single chain antibody to dsDNA was shown by ELISA, and cellular penetration and nuclear localization were demonstrated in COS-7 and CHO cells. The single chain antibody cDNA was ligated into the expression vector, pEGFP, to produce a fusion protein with green fluorescent protein. The fusion protein penetrated COS-7 cells and localized in the cell nucleus. The single chain antibody produced during sustained expression in CHO cells re-entered antibody-producing cells and localized in the nucleus without affecting cell viability. Our results demonstrate the potential use of a modified autoantibody as a delivery system to target the cell nucleus.

MeSH terms

  • Animals
  • Antibodies, Antinuclear / administration & dosage*
  • Antibodies, Antinuclear / genetics
  • Antibodies, Antinuclear / therapeutic use*
  • Antibodies, Monoclonal / administration & dosage*
  • Antibodies, Monoclonal / genetics
  • Antibodies, Monoclonal / therapeutic use*
  • Base Sequence
  • CHO Cells
  • COS Cells
  • Cell Nucleus / immunology
  • Cell Nucleus / metabolism
  • Cricetinae
  • DNA Primers / genetics
  • Drug Delivery Systems*
  • Green Fluorescent Proteins
  • Immunoglobulin Fab Fragments / administration & dosage
  • Immunoglobulin Fab Fragments / genetics
  • Immunoglobulin Fab Fragments / metabolism
  • Immunoglobulin Heavy Chains / administration & dosage
  • Immunoglobulin Heavy Chains / genetics
  • Immunoglobulin Heavy Chains / metabolism
  • Luminescent Proteins / immunology
  • Mice
  • Point Mutation
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / immunology
  • Recombinant Fusion Proteins / therapeutic use

Substances

  • Antibodies, Antinuclear
  • Antibodies, Monoclonal
  • DNA Primers
  • Immunoglobulin Fab Fragments
  • Immunoglobulin Heavy Chains
  • Luminescent Proteins
  • Recombinant Fusion Proteins
  • Green Fluorescent Proteins