Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the wild-type cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelial cells. However, the limited efficacy of gene transfer vectors as well as extra- and intracellular barriers have prevented the development of a gene therapy-based treatment for CF. Here, we review the use of new viral and nonviral gene therapy vectors, as well as human artificial chromosomes, to overcome barriers to successful CFTR expression. Pre-clinical studies will surely benefit from novel animal models, such as CF pigs and ferrets. Prenatal gene therapy is a potential alternative to gene transfer to fully developed lungs. However, unresolved issues, including the possibility of adverse effects on pre- and postnatal development, the risk of initiating oncogenic or degenerative processes and germ line transmission require further investigation. Finally, we discuss the therapeutic potential of stem cells for CF lung disease. (C) 2011 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Gene and cell therapy for cystic fibrosis: From bench to bedside / Massimo, Conese; Ascenzioni, Fiorentina; A., Christopher Boyd; Charles, Coutelle; Ida De, Fino; Stefaan De, Smedt; Joanna, Rejman; Joseph, Rosenecker; Dirk, Schindelhauer; Bob J., Scholte. - In: JOURNAL OF CYSTIC FIBROSIS. - ISSN 1569-1993. - ELETTRONICO. - 10:2(2011), pp. S114-S128. [10.1016/s1569-1993(11)60017-9]

Gene and cell therapy for cystic fibrosis: From bench to bedside

ASCENZIONI, Fiorentina;
2011

Abstract

Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the wild-type cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelial cells. However, the limited efficacy of gene transfer vectors as well as extra- and intracellular barriers have prevented the development of a gene therapy-based treatment for CF. Here, we review the use of new viral and nonviral gene therapy vectors, as well as human artificial chromosomes, to overcome barriers to successful CFTR expression. Pre-clinical studies will surely benefit from novel animal models, such as CF pigs and ferrets. Prenatal gene therapy is a potential alternative to gene transfer to fully developed lungs. However, unresolved issues, including the possibility of adverse effects on pre- and postnatal development, the risk of initiating oncogenic or degenerative processes and germ line transmission require further investigation. Finally, we discuss the therapeutic potential of stem cells for CF lung disease. (C) 2011 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.
cystic fibrosis; stem cells; gene delivery; animal model; vector development; human artificial chromosome; in utero gene therapy; cftr
01 Pubblicazione su rivista::01a Articolo in rivista
Gene and cell therapy for cystic fibrosis: From bench to bedside / Massimo, Conese; Ascenzioni, Fiorentina; A., Christopher Boyd; Charles, Coutelle; Ida De, Fino; Stefaan De, Smedt; Joanna, Rejman; Joseph, Rosenecker; Dirk, Schindelhauer; Bob J., Scholte. - In: JOURNAL OF CYSTIC FIBROSIS. - ISSN 1569-1993. - ELETTRONICO. - 10:2(2011), pp. S114-S128. [10.1016/s1569-1993(11)60017-9]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/417926
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