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.
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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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