A new Era of Personalized Medicine for Cystic Fibrosis – at Last!
The gene responsible for cystic fibrosis (CF) was discovered 25 years ago. This breakthrough has enabled a sophisticated understanding of how various mutations lead to specific alterations in the structure and function of the CF transmembrane regulator (CFTR) protein. Until recently, all therapies i...
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| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Wiley
2015-01-01
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| Series: | Canadian Respiratory Journal |
| Online Access: | http://dx.doi.org/10.1155/2015/921712 |
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| _version_ | 1849404986829570048 |
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| author | Bradley S Quon Pearce G Wilcox |
| author_facet | Bradley S Quon Pearce G Wilcox |
| author_sort | Bradley S Quon |
| collection | DOAJ |
| description | The gene responsible for cystic fibrosis (CF) was discovered 25 years ago. This breakthrough has enabled a sophisticated understanding of how various mutations lead to specific alterations in the structure and function of the CF transmembrane regulator (CFTR) protein. Until recently, all therapies in CF were focused on ameliorating the downstream consequences of CFTR dysfunction. High-throughput drug screening approaches have yielded compounds that can modify CFTR structure and function, thus targeting the basic defect in CF. The present article describes the CFTR mutational classes, reviews mutation-specific therapies currently in late-phase clinical development, and highlights research opportunities and challenges with personalized medicine in CF. |
| format | Article |
| id | doaj-art-48e7c668025e4cf0a27b35b0ea29adf6 |
| institution | Kabale University |
| issn | 1198-2241 |
| language | English |
| publishDate | 2015-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Canadian Respiratory Journal |
| spelling | doaj-art-48e7c668025e4cf0a27b35b0ea29adf62025-08-20T03:36:48ZengWileyCanadian Respiratory Journal1198-22412015-01-0122525726010.1155/2015/921712A new Era of Personalized Medicine for Cystic Fibrosis – at Last!Bradley S Quon0Pearce G Wilcox1Centre for Heart Lung Innovation, University of British Columbia and St Paul’s Hospital, CanadaCentre for Heart Lung Innovation, University of British Columbia and St Paul’s Hospital, CanadaThe gene responsible for cystic fibrosis (CF) was discovered 25 years ago. This breakthrough has enabled a sophisticated understanding of how various mutations lead to specific alterations in the structure and function of the CF transmembrane regulator (CFTR) protein. Until recently, all therapies in CF were focused on ameliorating the downstream consequences of CFTR dysfunction. High-throughput drug screening approaches have yielded compounds that can modify CFTR structure and function, thus targeting the basic defect in CF. The present article describes the CFTR mutational classes, reviews mutation-specific therapies currently in late-phase clinical development, and highlights research opportunities and challenges with personalized medicine in CF.http://dx.doi.org/10.1155/2015/921712 |
| spellingShingle | Bradley S Quon Pearce G Wilcox A new Era of Personalized Medicine for Cystic Fibrosis – at Last! Canadian Respiratory Journal |
| title | A new Era of Personalized Medicine for Cystic Fibrosis – at Last! |
| title_full | A new Era of Personalized Medicine for Cystic Fibrosis – at Last! |
| title_fullStr | A new Era of Personalized Medicine for Cystic Fibrosis – at Last! |
| title_full_unstemmed | A new Era of Personalized Medicine for Cystic Fibrosis – at Last! |
| title_short | A new Era of Personalized Medicine for Cystic Fibrosis – at Last! |
| title_sort | new era of personalized medicine for cystic fibrosis at last |
| url | http://dx.doi.org/10.1155/2015/921712 |
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