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...

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Bibliographic Details
Main Authors: Castellani, Stefano, Conese, Massimo
Format: Online
Language:English
Published: Molecular Diversity Preservation International (MDPI) 2010
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3185599/