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Genetic therapies for cystic fibrosis lung disease

Hart, SL; Harrison, PT; (2017) Genetic therapies for cystic fibrosis lung disease. Current Opinion in Pharmacology , 34 pp. 119-124. 10.1016/j.coph.2017.10.006. Green open access

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Abstract

Gene therapy for cystic fibrosis (CF) has been the subject ofintense research over the last twenty-five years or more, usingboth viral and liposomal delivery methods, but so far withoutthe emergence of a clinical therapy. New approaches to CFgene therapy involving recent improvements to vector systems,both viral and non-viral, as well as new nucleic acidtechnologies have led to renewed interest in the field. The fieldof therapeutic gene editing is rapidly developing with theemergence of CRISPR/Cas9 as well as chemically modifiedmRNA therapeutics. These new types of nucleic acid therapiesare also a good fit with delivery by non-viral deliveryapproaches which has led to a renewed interest in lipid-basedand other nanoformulations

Type: Article
Title: Genetic therapies for cystic fibrosis lung disease
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.coph.2017.10.006
Publisher version: http://doi.org/10.1016/j.coph.2017.10.006
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.
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/10048264
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