- 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 ...
Expand abstract - Publication status:
- Published
- Peer review status:
- Peer reviewed
- Publisher copy:
- 10.1002/ppul.23543
-
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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
- Language:
- English
- Keywords:
- Copyright date:
- 2016
Journal article
Genetic medicines for CF: Hype versus reality
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National Institute for Health Research
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Chief Scientist's Office in Scotland
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National Institute for Social Care and Health Research
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