| Gene and cell therapy for cystic fibrosis: From bench to bedside. | |
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MedLine Citation:
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PMID: 21658631 Owner: NLM Status: In-Data-Review |
Abstract/OtherAbstract:
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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. |
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Authors:
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Massimo Conese; Fiorentina Ascenzioni; A Christopher Boyd; Charles Coutelle; Ida De Fino; Stefaan De Smedt; Joanna Rejman; Joseph Rosenecker; Dirk Schindelhauer; Bob J Scholte |
Publication Detail:
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Type: Journal Article |
Journal Detail:
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Title: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society Volume: 10 Suppl 2 ISSN: 1873-5010 ISO Abbreviation: J. Cyst. Fibros. Publication Date: 2011 Jun |
Date Detail:
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Created Date: 2011-06-10 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 101128966 Medline TA: J Cyst Fibros Country: Netherlands |
Other Details:
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Languages: eng Pagination: S114-28 Citation Subset: IM |
Copyright Information:
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Copyright © 2011 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved. |
Affiliation:
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Institute for the Experimental Treatment of Cystic Fibrosis, H.S. Raffaele, 20132 Milan, Italy; Department of Biomédical Sciences, University of Foggia, 71100 Foggia, Italy. |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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