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In Vitro And In Vivo Functional Characterization Of Gutless Recombinant Sv40-Derived Cftr Vectors, Christian Mueller, M. Strayer, Jeffrey Sirninger, Sofia Braag, Francisco Branco, Jean-Pierre Louboutin, Terence Flotte, David Strayer Mar 2012

In Vitro And In Vivo Functional Characterization Of Gutless Recombinant Sv40-Derived Cftr Vectors, Christian Mueller, M. Strayer, Jeffrey Sirninger, Sofia Braag, Francisco Branco, Jean-Pierre Louboutin, Terence Flotte, David Strayer

Christian Mueller

In cystic fibrosis (CF), respiratory failure caused by progressive airway obstruction and tissue damage is primarily a result of the aberrant inflammatory responses to lung infections with Pseudomonas aeruginosa. Despite considerable improvement in patient survival, conventional therapies are mainly supportive. Recent progress toward gene therapy for CF has been encouraging; however, several factors such as immune response and transduced cell turnover remain as potential limitations to CF gene therapy. As alternative gene therapy vectors for CF, we examined the feasibility of using recombinant SV40-derived vectors (rSV40s), which may circumvent some of these obstacles. To accommodate the large cystic fibrosis transmembrane …