期刊
JOURNAL OF CYSTIC FIBROSIS
卷 10, 期 -, 页码 S114-S128出版社
ELSEVIER
DOI: 10.1016/S1569-1993(11)60017-9
关键词
Cystic fibrosis; CFTR; Human artificial chromosome; Animal model; Vector development; Gene delivery; In utero gene therapy; Stem cells
资金
- European Union [LSHM-CT-2005-018932]
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.
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