Journal Article

Genetic therapies for cystic fibrosis lung disease

Patrick L. Sinn, Reshma M. Anthony and Paul B. McCray

in Human Molecular Genetics

Volume 20, issue R1, pages R79-R86
Published in print April 2011 | ISSN: 0964-6906
Published online March 2011 | e-ISSN: 1460-2083 | DOI: http://dx.doi.org/10.1093/hmg/ddr104
Genetic therapies for cystic fibrosis lung disease

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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.

Journal Article.  4984 words.  Illustrated.

Subjects: Genetics and Genomics

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