Genetic Medicines for CF: Hype versus Reality
File(s)
Accepted version
Author(s)
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.
Date Issued
2016-09-23
Date Acceptance
2016-07-04
Citation
Pediatric Pulmonology, 2016, 51 (Supplement S44), pp.S5-S17
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
Medical Research Council (MRC)
National Institute for Health Research
Grant Number
MR/J014699/1
11/14/25
Subjects
cystic fibrosis (CF)
gene therapy
genome editing
lung
Respiratory System
1114 Paediatrics And Reproductive Medicine
Publication Status
Published