Lentiviral Vectors and Cystic Fibrosis Gene Therapy
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 th...
| Main Authors: | , |
|---|---|
| Format: | Online |
| Language: | English |
| Published: |
Molecular Diversity Preservation International (MDPI)
2010
|
| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3185599/ |
| id |
pubmed-3185599 |
|---|---|
| recordtype |
oai_dc |
| spelling |
pubmed-31855992011-10-12 Lentiviral Vectors and Cystic Fibrosis Gene Therapy Castellani, Stefano Conese, Massimo Review 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. Molecular Diversity Preservation International (MDPI) 2010-01-29 /pmc/articles/PMC3185599/ /pubmed/21994643 http://dx.doi.org/10.3390/v2020395 Text en © 2010 by the authors; licensee Molecular Diversity Preservation International, Basel, Switzerland. http://creativecommons.org/licenses/by/3.0 This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution license (http://creativecommons.org/licenses/by/3.0/). |
| repository_type |
Open Access Journal |
| institution_category |
Foreign Institution |
| institution |
US National Center for Biotechnology Information |
| building |
NCBI PubMed |
| collection |
Online Access |
| language |
English |
| format |
Online |
| author |
Castellani, Stefano Conese, Massimo |
| spellingShingle |
Castellani, Stefano Conese, Massimo Lentiviral Vectors and Cystic Fibrosis Gene Therapy |
| author_facet |
Castellani, Stefano Conese, Massimo |
| author_sort |
Castellani, Stefano |
| title |
Lentiviral Vectors and Cystic Fibrosis Gene Therapy |
| title_short |
Lentiviral Vectors and Cystic Fibrosis Gene Therapy |
| title_full |
Lentiviral Vectors and Cystic Fibrosis Gene Therapy |
| title_fullStr |
Lentiviral Vectors and Cystic Fibrosis Gene Therapy |
| title_full_unstemmed |
Lentiviral Vectors and Cystic Fibrosis Gene Therapy |
| title_sort |
lentiviral vectors and cystic fibrosis gene therapy |
| description |
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. |
| publisher |
Molecular Diversity Preservation International (MDPI) |
| publishDate |
2010 |
| url |
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3185599/ |
| _version_ |
1611479070529290240 |