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Research centres to revolutionise cystic fibrosis care

6 March 2015

The development of new drugs to treat the most common cause of cystic fibrosis, a genetic defect (or mutation) carried by around 90 per cent of people living with the disease in the UK, will be helped by the creation of a new virtual Strategic Research Centre (SRC) led by the University of Bristol.

The F508del-CFTR Strategic Research Centre, brings together scientists from Bristol, Manchester, University College London, Utrecht (the Netherlands) and Poitiers (France).

The centre is one of two new virtual Strategic Research Centres (SRC) of excellence awarded by the Cystic Fibrosis Trust to bring together the brightest and best scientists from across the UK and beyond to tackle specific problems within the field of cystic fibrosis.

In cystic fibrosis, CFTR, the protein that goes wrong in CF forms a gated pathway for chloride, one part of salt, to cross cell borders lining ducts and tubes throughout the body. The most common mutation in the CFTR gene, F508del, prevents CFTR proteins assembling correctly within the cell. The F508del-CFTR SRC will investigate the structure of CFTR to learn how it is affected by F508del, and search for chemicals that repair all the faults caused by the mutation. This would enable CFTR to be correctly made and delivered to the cell border.

Dr David Sheppard, who is Bristol's SRC lead and is based at the School of Physiology and Pharmacology, said: "This new centre provides a unique opportunity for a group of cystic fibrosis scientists to tackle together the inherited defect that is the most frequent root cause of the disease. By working together we will better inform the development of transformational drug therapies and train the next generation of cystic fibrosis scientists."

The second SRC 'the Cystic Fibrosis Epidemiological Network (CF-EpiNet)' will be led by Professor Di Bilton of the Royal Brompton Hospital, London. This SRC involves experts from the UK and Canada. Together, they will explore harnessing patient data to improve the care of people living with cystic fibrosis.

The team will extend the range and use of UK CF Registry data, which contains information for over 99 per cent of the UK CF population, linking it with other health and educational records to help identify treatments that most benefit health, survival and quality-of-life at critical life stages.

Research centres to revolutionise cystic fibrosis care
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