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Hum Mol Genet. 2011 Apr 15;20(R1):R79-86. doi: 10.1093/hmg/ddr104. Epub 2011 Mar 21.

Genetic therapies for cystic fibrosis lung disease.

Author information

  • 1Program in Gene Therapy, Department of Pediatrics, Carver College of Medicine, The University of Iowa, Iowa City, IA 52242, USA.

Abstract

The aim of gene therapy for cystic fibrosis (CF) lung disease is to efficiently and safely express the CF transmembrane conductance regulator (CFTR) in the appropriate pulmonary cell types. Although CF patients experience multi-organ disease, the chronic bacterial lung infections and associated inflammation are the primary cause of shortened life expectancy. Gene transfer-based therapeutic approaches are feasible, in part, because the airway epithelium is directly accessible by aerosol delivery or instillation. Improvements in standard delivery vectors and the development of novel vectors, as well as emerging technologies and new animal models, are propelling exciting new research forward. Here, we review recent developments that are advancing this field of investigation.

PMID:
21422098
[PubMed - indexed for MEDLINE]
PMCID:
PMC3095059
Free PMC Article
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