Chapter 4. Receptor-Directed Gene Delivery Using Molecular Conjugates

  1. David T. Curiel M.D. and
  2. Joanne T. Douglas Ph.D.
  1. Assem G. Ziady Ph.D.1 and
  2. Pamela B. Davis M.D., Ph.D.2

Published Online: 31 MAR 2003

DOI: 10.1002/0471234303.ch4

Vector Targeting for Therapeutic Gene Delivery

Vector Targeting for Therapeutic Gene Delivery

How to Cite

Ziady, A. G. and Davis, P. B. (2002) Receptor-Directed Gene Delivery Using Molecular Conjugates, in Vector Targeting for Therapeutic Gene Delivery (eds D. T. Curiel and J. T. Douglas), John Wiley & Sons, Inc., Hoboken, NJ, USA. doi: 10.1002/0471234303.ch4

Editor Information

  1. Division of Human Gene Therapy, Departments of Medicine, Pathology and Surgery, and the Gene Therapy Center, The University of Alabama at Birmingham, USA

Author Information

  1. 1

    Department of Pediatrics at Rainbow Babies and Childrens Hospital, Case Western Reserve University School of Medicine, Cleveland, Ohio, USA

  2. 2

    Division of Pediatric Pulmonology, Rainbow Babies and Childrens Hospital, Case Western Reserve University School of Medicine, Cleveland, Ohio, USA

Publication History

  1. Published Online: 31 MAR 2003
  2. Published Print: 9 AUG 2002

ISBN Information

Print ISBN: 9780471434795

Online ISBN: 9780471234302

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Keywords:

  • poly-L-lysine;
  • polyethyleneimine;
  • receptor-directed gene transfer;
  • endosomal escape;
  • DNA compaction;
  • asialoglycoprotein receptor;
  • serpin enzyme complex receptor;
  • polymeric immunoglobulin receptor;
  • transferrin receptor;
  • epidermal growth factor receptor;
  • fibroblast growth factor receptor

Summary

In an effort to achieve delivery of therapeutic genes specifically to particular target cells, using a non-viral vector, molecular conjugates consisting of a receptor ligand, polycation to bind to DNA, and plasmid DNA have been constructed. Some successful complexes have capitalized on DNA compaction, which protects the plasmid from nuclease degradation and facilitates nuclear access in non-dividing cells. The chemical construction of the complex, including the density of the ligands, the nature of the polycation, the stabilizing agents used, as well as the specific receptor targeted and the ligands selected all affect the success of the gene transfer.