Gene therapy for B-cell lymphoma in a SCID mouse model using an immunoglobulin-regulated diphtheria toxin gene delivered by a novel adenovirus-polylysine conjugate

Cancer Biother. 1994 Summer;9(2):131-41. doi: 10.1089/cbr.1994.9.131.

Abstract

Despite advances in conventional therapy, many lives continue to be lost to common forms of B-cell cancers, including leukemias, lymphomas and multiple myeloma. We propose a novel approach to therapy of such cancers using controlled expression of a diphtheria toxin gene (DT-A) to kill malignant cells. We have previously demonstrated selective killing of various cell types, in vitro and in vivo, by cell-specific, transcriptionally controlled expression of this gene. Organ-specific ablation in otherwise healthy transgenic mice has convincingly demonstrated the exquisite specificity achievable by this technique. In the studies now described, DT-A was delivered in vitro and in vivo using a novel gene delivery system employing DNA physically attached to the exterior of adenovirus. After demonstrating the efficacy of gene delivery to Epstein-Barr virus transformed human B-cells in vitro, in vivo work was performed using a SCID mouse model for B-cell lymphoma, in which protection against tumor was observed. The concepts of tissue-regulated toxin gene therapy, and this novel adenovirus gene delivery system are discussed.

Publication types

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

MeSH terms

  • Adenoviridae*
  • Animals
  • Biotin
  • Cell Line, Transformed
  • DNA, Recombinant / administration & dosage*
  • Diphtheria Toxin / biosynthesis
  • Diphtheria Toxin / genetics
  • Diphtheria Toxin / therapeutic use*
  • Female
  • Gene Expression Regulation, Neoplastic
  • Genes, Immunoglobulin*
  • Genes, Reporter
  • Genes, Synthetic*
  • Genetic Therapy*
  • Herpesvirus 4, Human
  • Immunoglobulin kappa-Chains / genetics*
  • Ligands
  • Lymphoma, B-Cell / genetics
  • Lymphoma, B-Cell / immunology
  • Lymphoma, B-Cell / therapy*
  • Male
  • Mice
  • Mice, SCID
  • Peptide Fragments / biosynthesis
  • Peptide Fragments / genetics
  • Peptide Fragments / therapeutic use*
  • Polylysine*
  • Promoter Regions, Genetic*
  • Recombinant Fusion Proteins / biosynthesis
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / therapeutic use

Substances

  • DNA, Recombinant
  • Diphtheria Toxin
  • Immunoglobulin kappa-Chains
  • Ligands
  • Peptide Fragments
  • Recombinant Fusion Proteins
  • diphtheria toxin fragment A
  • Polylysine
  • Biotin