Journal
MOLECULAR THERAPY
Volume 20, Issue 6, Pages 1108-1115Publisher
CELL PRESS
DOI: 10.1038/mt.2012.32
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Funding
- NIH [P01 HL-51670, P01 HL-091842]
- Roy J. Carver Charitable Trust
- In Vitro Models and Cell Culture Core
- Gene Transfer Vector Core
- Cell Morphology Core
- Center for Gene Therapy for Cystic Fibrosis [NIH P30 DK-54759]
- Cystic Fibrosis Foundation
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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.
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