Non-Viral Gene Therapy for Pulmonary Disease

  • Myra Stern
  • Eric Alton


Non-viral gene therapy now includes a number of different strategies ranging from direct intramuscular injection of naked DNA to the systemic or local administration of formulations comprising DNA and lipid, proteins, peptides or polymers. While the main limitation of non-viral gene transfer methods is their relatively low efficiency in vivo, both preclinical and clinical studies indicate that these methods exhibit safety profiles similar to conventional pharmaceutical and biological products. Cationic liposome-based gene therapy has been extensively studied for the treatment of the genetic disease cystic fibrosis, where research has reached the stage of clinical trials. Non-viral gene delivery has also been studied with a view to developing treatment for the acute lung injury and pulmonary oedema associated with the adult respiratory distress syndrome (ARDS). In this chapter, following a brief overview of non-viral gene transfer, the progress of both clinical studies for CF gene therapy and preclinical studies for pulmonary oedema are described.


Cystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator Airway Epithelial Cell Cationic Lipid Cationic Liposome 
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Copyright information

© Springer Science+Business Media New York 2001

Authors and Affiliations

  • Myra Stern
    • 1
  • Eric Alton
    • 1
  1. 1.Department of Gene TherapyImperial College at the National Heart and Lung InstituteLondonUK

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