Cystic Fibrosis Gene Therapy


Cystic Fibrosis Trust
The Cystic Fibrosis Trust is the UK's only national charity dealing with all aspects of Cystic Fibrosis. It funds research to treat the symptoms of CF and to find an effective treatment. It also aims to ensure appropriate healthcare for those with Cystic Fibrosis and provides information, advice and support to people with CF and their families across the UK. Cystic Fibrosis is one of the most common, life-threatening diseases in the UK. It affects over 9,000 people and 1 in 25 of us carries the faulty gene that causes Cystic Fibrosis. If two carriers start a family, each child has a 1 in 4 chance of having Cystic Fibrosis. Cystic Fibrosis affects the internal organs, especially the lungs and digestive system, by clogging them with thick, sticky mucus making it hard to breathe and to digest food. People with Cystic Fibrosis have to endure a rigorous daily treatment regime including physiotherapy to clear the chest, inhaled and injected drugs to thin the mucus and fight infections and dozens of enzyme pills to help digest food. Each week, five babies are born with CF and sadly, each week three young lives are lost. Only half of those with Cystic Fibrosis are likely to live past their late 30s, but a baby born today can expect to live longer. We seek to continually improve standards of care for people with CF; we commission cutting-edge clinical and scientific research; and provide practical and accurate information to people with CF and their families. None of this would be possible without the support of the CF community and the generosity of the public.

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Cystic Fibrosis Trust - Gene Therapy Appeal

Cystic Fibrosis Gene Therapy

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Following 10 years of development and successful tests on safety and toxicity over the last two years, a multi-dose clinical trial for CF gene therapy is planned to start in Spring 2012. 

The progress is a great tribute to those in the UK CF Gene Therapy Consortium whose work has been recognised across the world, and for the thousands of families and benefactors whose huge efforts have raised the £30 million the research has cost so far.

This is a crucial stage in the research but will cost a further £6m.  This appeal has been launched to ensure that the multi-dose trial goes ahead.  This is a lot of money and, of course, there are no guarantees of outcomes in this work. But the chance of securing a multi-dose trial is a once-in-a-lifetime opportunity which we cannot miss. Without proceeding with this trial we would not know whether effective gene therapy for CF could become a reality. So, we will be making every effort to raise the money required and looking for the support of all in the CF community and beyond.

This is the first such gene therapy trial in the world and has the potential to benefit the estimated 100,000 people with CF across the globe.

Recent donors









08.02.12 In memory of Rod Leaver RIP


28.01.12 in honour of Thomas and all he achieved, and in support of Ian and Annie at this most difficult time.

26.01.12 During a little game within the family, my brother-in-law won £5, and I've matched it to give to the CF Trust, my grandson Benjamin Mudge has CF, so I naturally want to help with research.

25.01.12 Raised by Chiddingstone Amateur Players Society - CHAPS - during their Christmas Production of Lady Fazerkely's Family Festive Feast.



22.01.12 Mary Knight Chiropody patient donations & other donations





10.01.12 donated from The 33, wishing you all the very best with your fundraising in 2012.

10.01.12 This donation was collected from the Heather Primary School Christmas Performances as our charity for the evdent

09.01.12 Hilary's birthday present


06.01.12 For our gorgeous Harry, with lots of love xxx


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