Genetic medicines for CF: Hype versus reality

被引：36

作者：

Alton, Eric W. F. W. ^{[1
]}

Boyd, A. Christopher ^{[1
]}

Davies, Jane C. ^{[1
]}

Gill, Deborah R. ^{[1
]}

Griesenbach, Uta ^{[1
]}

Harrison, Patrick T. ^{[2
,3
]}

Henig, Noreen ^{[4
]}

Higgins, Tracy ^{[1
]}

Hyde, Stephen C. ^{[1
]}

Innes, J. Alastair ^{[1
]}

Korman, Michael S. D. ^{[5
]}

机构：

[1] UK Cyst Fibrosis Gene Therapy Consortium, Edinburgh, Midlothian, Scotland

[2] Univ Coll Cork, Dept Physiol, Cork, Ireland

[3] Univ Coll Cork, BioSci Inst, Cork, Ireland

[4] ProQR Therapeut NV, Leiden, Netherlands

[5] Univ Tubingen, Dept Pediat Pediat Infectiol & Immunol Translat G, Tubingen, Germany

来源：

PEDIATRIC PULMONOLOGY | 2016年 / 51卷

基金：

英国医学研究理事会;

关键词：

cystic fibrosis (CF); gene therapy; lung; genome editing; NASAL POTENTIAL DIFFERENCE; PROLONGED TRANSGENE EXPRESSION; CONDUCTANCE REGULATOR GENE; PLACEBO-CONTROLLED TRIAL; ENCODING MESSENGER-RNA; PLURIPOTENT STEM-CELLS; ZINC-FINGER NUCLEASES; CYSTIC-FIBROSIS; LENTIVIRAL VECTOR; AIRWAY EPITHELIUM;

D O I：

10.1002/ppul.23543

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

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. (c) 2016 Wiley Periodicals, Inc.

引用

页码：S5 / S17

页数：13

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