Journal article
Gene therapy for respiratory diseases; progress and a changing context
- Abstract:
- The UK Respiratory Gene Therapy Consortium (GTC) The GTC was formed in 2001 from three groups at the Universities of Edinburgh and Oxford and Imperial College, London to explore gene therapy as a therapeutic option for people with cystic fibrosis (CF)1. The gene responsible for CF, Cystic Fibrosis Transmembrane conductance Regulator (CFTR), was identified in 19892 and over 2000 mutations are now known3, typically classified into six groups4. Whilst considerable progress has been made with this mutation-agnostic approach, gene therapy is not yet a clinical reality. In parallel, mutation-specific, small molecule CFTR modulator therapy has now demonstrated substantial clinical efficacy5. Here, we briefly summarise the opinions of the GTC on navigating this evolving terrain, as well as noting some opportunities for gene therapy in other respiratory diseases.
- Publication status:
- Published
- Peer review status:
- Peer reviewed
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Access Document
- Files:
-
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(Preview, Accepted manuscript, 625.3KB, Terms of use)
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- Publisher copy:
- 10.1089/hum.2020.142
Authors
- Publisher:
- Mary Ann Liebert
- Journal:
- Human Gene Therapy More from this journal
- Volume:
- 31
- Issue:
- 17-18
- Pages:
- 911-916
- Publication date:
- 2020-08-04
- Acceptance date:
- 2020-08-04
- DOI:
- EISSN:
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1557-7422
- ISSN:
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1043-0342
- Pmid:
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32746737
- Language:
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English
- Keywords:
- Pubs id:
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1126545
- Local pid:
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pubs:1126545
- Deposit date:
-
2020-08-20
Terms of use
- Copyright holder:
- Mary Ann Liebert, Inc., publishers
- Copyright date:
- 2020
- Rights statement:
- © 2020 Mary Ann Liebert, Inc., publishers. All rights reserved, USA and worldwide.
- Notes:
-
This is the accepted manuscript version of the article. The final version is available from Mary Ann Liebert at https://doi.org/10.1089/hum.2020.142
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