Design of a genetic immunotoxin to eliminate toxin immunogenicity

Gene Ther. 1995 Mar;2(2):116-23.

Abstract

Host antibody response to toxin molecules is a major obstacle to the use of immunotoxins as efficacious agents in the treatment of human cancer and other diseases. In this study, a genetic form of immunotoxin has been designed which should eliminate toxin immunogenicity by replacing the toxin protein moiety with weakly immunogenic or nonimmunogenic plasmid DNA. A recombinant bifunctional fusion protein, which consists of a human antibody Fab targeting moiety [directed against gp120, the envelope glycoprotein of human immunodeficiency virus (HIV)-1] and a human DNA binding moiety (protamine), is used as a gene carrier. Toxin plasmid DNAs expressing the catalytic fragment of Pseudomonas exotoxin A (PEA) statically interact with the fusion proteins to form soluble protein-DNA complexes. The complexes are specifically transferred into HIV-1-infected cells by receptor-mediated endocytosis, resulting in selective killing of the target cells. These 'genetic immunotoxins' may have significant advantages over protein immunotoxins for the treatment of a variety of human diseases.

Publication types

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

MeSH terms

  • ADP Ribose Transferases*
  • Animals
  • Antibodies, Monoclonal / immunology*
  • Bacterial Toxins*
  • Base Sequence
  • CD4-Positive T-Lymphocytes / drug effects
  • CD4-Positive T-Lymphocytes / virology
  • Cell Line, Transformed
  • Chlorocebus aethiops
  • Cytomegalovirus / genetics
  • DNA / metabolism
  • Drug Design
  • Exotoxins / administration & dosage*
  • Exotoxins / chemistry
  • Exotoxins / genetics
  • Exotoxins / toxicity
  • Genetic Vectors
  • HIV Antibodies / immunology*
  • HIV Envelope Protein gp120 / immunology*
  • HIV Envelope Protein gp120 / metabolism
  • HIV-1 / immunology*
  • Humans
  • Immunoglobulin Fab Fragments / immunology*
  • Immunoglobulin Heavy Chains / immunology*
  • Immunoglobulin Heavy Chains / metabolism
  • Immunotoxins / immunology*
  • Immunotoxins / metabolism
  • Molecular Sequence Data
  • Protamines / immunology*
  • Pseudomonas aeruginosa Exotoxin A
  • Recombinant Fusion Proteins / immunology*
  • Recombinant Fusion Proteins / metabolism
  • Tumor Cells, Cultured
  • Virulence Factors*

Substances

  • Antibodies, Monoclonal
  • Bacterial Toxins
  • Exotoxins
  • HIV Antibodies
  • HIV Envelope Protein gp120
  • Immunoglobulin Fab Fragments
  • Immunoglobulin Heavy Chains
  • Immunotoxins
  • Protamines
  • Recombinant Fusion Proteins
  • Virulence Factors
  • DNA
  • ADP Ribose Transferases