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New approaches to genetic therapies for cystic fibrosis

Boyd, AC; Guo, S; Huang, L; Kerem, B; Oren, YS; Walker, AJ; Hart, SL; (2020) New approaches to genetic therapies for cystic fibrosis. Journal of Cystic Fibrosis , 19 (S1) S54-S59. 10.1016/j.jcf.2019.12.012. Green open access

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Abstract

Gene therapy offers great promise for cystic fibrosis which has never been quite fulfilled due to the challenges of delivering sufficient amounts of the CFTR gene and expression persistence for a sufficient period of time in the lungs to have any effect. Initial trials explored both viral and non-viral vectors but failed to achieve a significant breakthrough. However, in recent years, new opportunities have emerged that exploit our increased knowledge and understanding of the biology of CF and the airway epithelium. New technologies include new viral and non-viral vector approaches to delivery, but also alternative nucleic acid technologies including oligonucleotides and siRNA approaches for gene silencing and gene splicing, described in this review, as presented at the 2019 annual European CF Society Basic Science meeting (Dubrovnik, Croatia). We also briefly discuss other emerging technologies including mRNA and CRISPR gene editing that are advancing rapidly. The future prospects for genetic therapies for CF are now diverse and more promising probably than any time since the discovery of the CF gene.

Type: Article
Title: New approaches to genetic therapies for cystic fibrosis
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.jcf.2019.12.012
Publisher version: https://doi.org/10.1016/j.jcf.2019.12.012
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Cystic fibrosis, Gene therapy, Antisense oligonucleotides, siRNA, mRNA splicing, ENaC, CFTR, Liposomes, Nanoparticles
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10090183
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