Genetic medicines for CF: hype versus reality

Show simple item record Alton, Eric W. F. W. Boyd, A. Christopher Davies, Jane C. Gill, Deborah R. Griesenbach, Uta Harrison, Patrick T. Henig, Noreen Higgins, Tracy Hyde, Stephen C. Innes, J. Alastair Korman, Michael S. D. 2016-10-04T14:07:13Z 2016-10-04T14:07:13Z 2016-09-23
dc.identifier.citation Alton, E. W. F. W., Boyd, A. C., Davies, J. C., Gill, D. R., Griesenbach, U., Harrison, P. T., Henig, N., Higgins, T., Hyde, S. C., Innes, J. A. & Korman, M. S. D. (2016) ‘Genetic medicines for CF: hype versus reality’, Pediatric Pulmonology, 51(S44), pp. S5-S17. doi: 10.1002/ppul.23543 en
dc.identifier.volume 51 en
dc.identifier.issued Supplement S44 en
dc.identifier.startpage S5 en
dc.identifier.endpage S17 en
dc.identifier.issn 8755-6863
dc.identifier.doi 10.1002/ppul.23543
dc.description.abstract Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of “genetic medicines” including mRNA delivery, as well as genome editing and mRNA repair-based strategies. Proof-of-concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editing-based strategies are currently at the pre-clinical phase of development. This review has been written jointly by some of those involved in the various CF “genetic medicine” fields and will summarize the current state-of-the-art, as well as discuss future developments. Where applicable, it highlights common problems faced by each of the strategies, and also tries to highlight where a specific strategy may have an advantage on the pathway to clinical translation. We hope that this review will contribute to the ongoing discussion about the hype versus reality of genetic medicine-based treatment approaches in CF. en
dc.description.sponsorship National Institute for Health Research, United Kingdom and Medical Research Council, United Kingdom(Efficacy and Mechanism Evaluation program (; Medical Research Council, United Kingdom (MRC DPFS program); National Institute for Health Research (NIHR Respiratory Biomedical Research Unit at the Royal Brompton and Harefield NHS Foundation Trust and the Edinburgh Clinical Research Facility at the Western General Hospital (NHS Lothian)). European Commission (European Research Council); en
dc.format.mimetype application/pdf en
dc.language.iso en en
dc.publisher Wiley en
dc.rights © 2016 Wiley Periodicals, Inc. This is the peer reviewed version of the following article: Alton, et al., ‘Genetic medicines for CF: hype versus reality’, Pediatr Pulmonol., 51: S5–S17, which has been published in final form at This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Self-Archiving. en
dc.subject Cystic fibrosis (CF) en
dc.subject Gene therapy en
dc.subject Genome editing en
dc.subject Lung en
dc.title Genetic medicines for CF: hype versus reality en
dc.type Article (peer-reviewed) en
dc.internal.authorcontactother Patrick Harrison, Physiology, University College Cork, Cork, Ireland. +353-21-490-3000 Email: en
dc.internal.availability Full text available en 12 month embargo at publisher's request. en 2017-09-23
dc.description.version Accepted Version en
dc.contributor.funder National Institute for Health Research, United Kingdom en
dc.contributor.funder Medical Research Council, United Kingdom en
dc.contributor.funder Chief Scientist Office, Scotland en
dc.contributor.funder National Institute for Social Care and Health Research (NISCHR), Wales en
dc.contributor.funder HSC Research and Development, Public Health Agency in Northern Ireland en
dc.contributor.funder The Cystic Fibrosis Trust, United Kingdom en
dc.contributor.funder Cystic Fibrosis Foundation, United Kingdom en
dc.contributor.funder Just Gene Therapy, United Kingdom en
dc.contributor.funder ERANDA foundation, United Kingdom en
dc.contributor.funder German Research Foundation en
dc.contributor.funder European Commission en
dc.description.status Peer reviewed en
dc.identifier.journaltitle Pediatric Pulmonology en
dc.internal.copyrightchecked !!CORA!! en
dc.internal.IRISemailaddress en

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