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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Authors
Funding
National Institute for Health Research
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National Institute for Social Care and Health Research
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Chief Scientist's Office in Scotland
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Bibliographic Details
- 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
- Pmid:
-
27662105
- Source identifiers:
-
648042
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:648042
- UUID:
-
uuid:4dd3e932-cf31-4d85-84db-ca71e932c7a0
- Local pid:
- pubs:648042
- Deposit date:
- 2018-08-06
Terms of use
- Copyright date:
- 2016
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