Cell and gene based therapies for Cystic Fibrosis lung disease

http://www.ncbi.nlm.nih.gov/pubmed/22371844

Mol Ther. 2012 Feb 28. doi: 10.1038/mt.2012.32. [Epub ahead of print]
Advances in Cell and Gene-based Therapies for Cystic Fibrosis Lung Disease.
Oakland M, Sinn PL, McCray Jr PB.
Source
Department of Microbiology, Carver College of Medicine, The University of Iowa, Iowa City, Iowa, USA.

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.
PMID: 22371844 [PubMed - as supplied by publisher]