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Curr Opin Pharmacol. 2017 Jun;34:119-124. doi: 10.1016/j.coph.2017.10.006. Epub 2017 Nov 5.

Genetic therapies for cystic fibrosis lung disease.

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Experimental and Personalised Medicines Section, Genetics and Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, 30 Guilford St, London WC1N 1EH, UK. Electronic address:
Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland.


Gene therapy for cystic fibrosis (CF) has been the subject of intense research over the last twenty-five years or more, using both viral and liposomal delivery methods, but so far without the emergence of a clinical therapy. New approaches to CF gene therapy involving recent improvements to vector systems, both viral and non-viral, as well as new nucleic acid technologies have led to renewed interest in the field. The field of therapeutic gene editing is rapidly developing with the emergence of CRISPR/Cas9 as well as chemically modified mRNA therapeutics. These new types of nucleic acid therapies are also a good fit with delivery by non-viral delivery approaches which has led to a renewed interest in lipid-based and other nanoformulations.

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