Challenges and strategies for cystic fibrosis lung gene therapy

Citation
Dr. Koehler et al., Challenges and strategies for cystic fibrosis lung gene therapy, MOL THER, 4(2), 2001, pp. 84-91
Citations number
54
Language
INGLESE
art.tipo
Review
Categorie Soggetti
Molecular Biology & Genetics
Journal title
MOLECULAR THERAPY
ISSN journal
1525-0016 → ACNP
Volume
4
Issue
2
Year of publication
2001
Pages
84 - 91
Database
ISI
SICI code
1525-0016(200108)4:2<84:CASFCF>2.0.ZU;2-V
Abstract
Gene replacement therapy represents an interesting new approach for the tre atment of cystic fibrosis (CF) lung disease. Basic research suggests that C F gene therapy is feasible, but major technological challenges must be addr essed before clinical applications are likely to succeed. Therapeutic genes can be delivered to and expressed in human airways, but the number of cell s expressing the transgene is relatively low. The inefficiency of gene deli very is largely attributable to the remarkable defenses of human airways. M aintaining long-term transgene expression in airway cells is also a signifi cant obstacle. Recent advances have been made in the development of vectors , expression cassettes, and delivery techniques for enhancing airway gene t ransfer and expression. These advances have the potential to improve the ef ficiency of lung gene therapy and to achieve clinical benefits for CF patie nts in the future.