Journal article
Genetic therapies for cystic fibrosis lung disease
Human molecular genetics, Vol.20(R1), pp.R79-R86
04/15/2011
DOI: 10.1093/hmg/ddr104
PMCID: PMC3095059
PMID: 21422098
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.
Details
- Title: Subtitle
- Genetic therapies for cystic fibrosis lung disease
- Creators
- Patrick L Sinn - Program in Gene Therapy, Department of Pediatrics, Carver College of Medicine, The University of Iowa, Iowa City, IA 52242, USAReshma M AnthonyPaul B McCray Jr
- Resource Type
- Journal article
- Publication Details
- Human molecular genetics, Vol.20(R1), pp.R79-R86
- DOI
- 10.1093/hmg/ddr104
- PMID
- 21422098
- PMCID
- PMC3095059
- ISSN
- 0964-6906
- eISSN
- 1460-2083
- Grant note
- P01 HL-51670 / NHLBI NIH HHS R01 HL-075363 / NHLBI NIH HHS P30 DK-54759 / NIDDK NIH HHS P30 DK054759 / NIDDK NIH HHS R21 HL-91808 / NHLBI NIH HHS
- Language
- English
- Date published
- 04/15/2011
- Academic Unit
- Microbiology and Immunology; Pulmonary Medicine; Stead Family Department of Pediatrics; Internal Medicine
- Record Identifier
- 9984093347202771
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