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Journal article

Genetic medicines for CF: Hype versus reality

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 ...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1002/ppul.23543

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Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
RDM; RDM Clinical Laboratory Sciences
Oxford college:
University College
Role:
Author
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National Institute for Health Research More from this funder
Chief Scientist's Office in Scotland More from this funder
National Institute for Social Care and Health Research More from this funder
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Publisher:
Wiley Publisher's website
Journal:
Pediatric Pulmonology Journal website
Volume:
51
Issue:
S44
Pages:
S5-S17
Publication date:
2016-09-23
Acceptance date:
2016-06-15
DOI:
EISSN:
1099-0496
ISSN:
8755-6863
Pubs id:
pubs:648042
URN:
uri:4dd3e932-cf31-4d85-84db-ca71e932c7a0
UUID:
uuid:4dd3e932-cf31-4d85-84db-ca71e932c7a0
Local pid:
pubs:648042

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