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Viruses 2010, 2(2), 395-412; doi:10.3390/v2020395

Lentiviral Vectors and Cystic Fibrosis Gene Therapy

Department of Biomedical Sciences, University of Foggia, Foggia, Italy
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Author to whom correspondence should be addressed.
Received: 7 October 2009 / Revised: 26 January 2010 / Accepted: 27 January 2010 / Published: 29 January 2010
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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Abstract

Cystic fibrosis (CF) is a chronic autosomic recessive syndrome, caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene, a chloride channel expressed on the apical side of the airway epithelial cells. The lack of CFTR activity brings a dysregulated exchange of ions and water through the airway epithelium, one of the main aspects of CF lung disease pathophysiology. Lentiviral (LV) vectors, of the Retroviridae family, show interesting properties for CF gene therapy, since they integrate into the host genome and allow long-lasting gene expression. Proof-of-principle that LV vectors can transduce the airway epithelium and correct the basic electrophysiological defect in CF mice has been given. Initial data also demonstrate that LV vectors can be repeatedly administered to the lung and do not give rise to a gross inflammatory process, although they can elicit a T cell-mediated response to the transgene. Future studies will clarify the efficacy and safety profile of LV vectors in new complex animal models with CF, such as ferrets and pigs.
Keywords: airway epithelium; cystic fibrosis transmembrane conductance regulator (CFTR); lentivirus; lung; Retroviridae airway epithelium; cystic fibrosis transmembrane conductance regulator (CFTR); lentivirus; lung; Retroviridae
This is an open access article distributed under the Creative Commons Attribution License (CC BY 3.0).

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Castellani, S.; Conese, M. Lentiviral Vectors and Cystic Fibrosis Gene Therapy. Viruses 2010, 2, 395-412.

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