Advances in cell and gene-based therapies for cystic fibrosis lung disease

Mol Ther. 2012 Jun;20(6):1108-15. doi: 10.1038/mt.2012.32. Epub 2012 Feb 28.


Cystic fibrosis (CF) is a disease characterized by airway infection, inflammation, remodeling, and obstruction that gradually destroy the lungs. Direct delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelia may offer advantages, as the tissue is accessible for topical delivery of vectors. Yet, physical and host immune barriers in the lung present challenges for successful gene transfer to the respiratory tract. Advances in gene transfer approaches, tissue engineering, and novel animal models are generating excitement within the CF research field. This review discusses current challenges and advancements in viral and nonviral vectors, cell-based therapies, and CF animal models.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Cystic Fibrosis / genetics
  • Cystic Fibrosis / therapy*
  • Disease Models, Animal
  • Genetic Therapy* / methods
  • Humans
  • Stem Cell Transplantation*
  • Tissue Transplantation