Genetic medicines for CF: hype versus reality

dc.check.info12 month embargo at publisher's request.en
dc.contributor.authorAlton, Eric W. F. W.
dc.contributor.authorBoyd, A. Christopher
dc.contributor.authorDavies, Jane C.
dc.contributor.authorGill, Deborah R.
dc.contributor.authorGriesenbach, Uta
dc.contributor.authorHarrison, Patrick T.
dc.contributor.authorHenig, Noreen
dc.contributor.authorHiggins, Tracy
dc.contributor.authorHyde, Stephen C.
dc.contributor.authorInnes, J. Alastair
dc.contributor.authorKorman, Michael S. D.
dc.contributor.funderNational Institute for Health Research, United Kingdomen
dc.contributor.funderMedical Research Council, United Kingdomen
dc.contributor.funderChief Scientist Office, Scotlanden
dc.contributor.funderNational Institute for Social Care and Health Research (NISCHR), Walesen
dc.contributor.funderHSC Research and Development, Public Health Agency in Northern Irelanden
dc.contributor.funderThe Cystic Fibrosis Trust, United Kingdomen
dc.contributor.funderCystic Fibrosis Foundation, United Kingdomen
dc.contributor.funderJust Gene Therapy, United Kingdomen
dc.contributor.funderERANDA foundation, United Kingdomen
dc.contributor.funderGerman Research Foundationen
dc.contributor.funderEuropean Commissionen
dc.description.abstractSince 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.sponsorshipNational 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.description.statusPeer revieweden
dc.description.versionAccepted Versionen
dc.identifier.citationAlton, 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.23543en
dc.identifier.issuedSupplement S44en
dc.identifier.journaltitlePediatric Pulmonologyen
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.subjectCystic fibrosis (CF)en
dc.subjectGene therapyen
dc.subjectGenome editingen
dc.titleGenetic medicines for CF: hype versus realityen
dc.typeArticle (peer-reviewed)en
Original bundle
Now showing 1 - 1 of 1
Thumbnail Image
1.02 MB
Adobe Portable Document Format
Accepted Version
License bundle
Now showing 1 - 1 of 1
Thumbnail Image
2.71 KB
Item-specific license agreed upon to submission