Journal article
Advances in cell and gene-based therapies for cystic fibrosis lung disease
Molecular therapy, Vol.20(6), pp.1108-1115
06/2012
DOI: 10.1038/mt.2012.32
PMCID: PMC3369283
PMID: 22371844
Abstract
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.
Details
- Title: Subtitle
- Advances in cell and gene-based therapies for cystic fibrosis lung disease
- Creators
- Mayumi Oakland - Department of Microbiology, Carver College of Medicine, The University of Iowa, Iowa City, Iowa 52242, USAPatrick L SinnPaul B McCray Jr
- Resource Type
- Journal article
- Publication Details
- Molecular therapy, Vol.20(6), pp.1108-1115
- DOI
- 10.1038/mt.2012.32
- PMID
- 22371844
- PMCID
- PMC3369283
- ISSN
- 1525-0016
- eISSN
- 1525-0024
- Grant note
- P01 HL-51670 / NHLBI NIH HHS NIH P30 DK-54759 / NIDDK NIH HHS P01 HL051670 / NHLBI NIH HHS P30 DK054759 / NIDDK NIH HHS P01 HL- 091842 / NHLBI NIH HHS
- Language
- English
- Date published
- 06/2012
- Academic Unit
- Microbiology and Immunology; Pulmonary Medicine; Stead Family Department of Pediatrics; Internal Medicine
- Record Identifier
- 9984093351702771
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