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Genetic Medicines for CF: Hype versus Reality

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NACF reveiw E Alton May 2016 clean version.docxAccepted version1.51 MBMicrosoft WordView/Open
Title: Genetic Medicines for CF: Hype versus Reality
Authors: Alton, EWFW
Boyd, AC
Davies, JC
Gill, DR
Griesenbach, U
Harrison, PT
Henig, N
Higgins, T
Hyde, SC
Innes, JA
Korman, MSD
Item Type: Journal Article
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.
Issue Date: 23-Sep-2016
Date of Acceptance: 4-Jul-2016
URI: http://hdl.handle.net/10044/1/39001
DOI: https://dx.doi.org/10.1002/ppul.23543
ISSN: 8755-6863
Publisher: Wiley
Start Page: S5
End Page: S17
Journal / Book Title: Pediatric Pulmonology
Volume: 51
Issue: Supplement S44
Copyright Statement: This is the peer reviewed version of the following article: 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. and Korman, M. S.D. (2016), Genetic medicines for CF: Hype versus reality. Pediatr Pulmonol., 51: S5–S17. which has been published in final form at https://dx.doi.org/10.1002/ppul.23543.  This article may be used for non-commercial purposes in accordance With Wiley Terms and Conditions for self-archiving.
Sponsor/Funder: Medical Research Council (MRC)
National Institute for Health Research
Funder's Grant Number: MR/J014699/1
11/14/25
Keywords: cystic fibrosis (CF)
gene therapy
genome editing
lung
Respiratory System
1114 Paediatrics And Reproductive Medicine
Publication Status: Published
Appears in Collections:National Heart and Lung Institute