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Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.

Genetic medicines for CF: Hype versus reality.

Author information

1
UK Cystic Fibrosis Gene Therapy Consortium, Edinburgh, Oxford, London.
2
UK Cystic Fibrosis Gene Therapy Consortium, Edinburgh, Oxford, London. u.griesenbach@imperial.ac.uk.
3
Department of Physiology and BioSciences Institute, University College Cork, Cork, Ireland.
4
ProQR Therapeutics NV, Leiden, The Netherlands.
5
Department of Pediatrics I - Pediatric Infectiology and Immunology - Translational Genomics and Gene Therapy, University of Tübingen, Tübingen, Germany.

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. Pediatr Pulmonol. 2016;51:S5-S17.

KEYWORDS:

cystic fibrosis (CF); gene therapy; genome editing; lung

PMID:
27662105
DOI:
10.1002/ppul.23543
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