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CF Gene Therapy Research at the University of Edinburgh. Cystic fibrosis (CF) is a common inherited life-threatening disease. About 1 in 25 people is a carrier; there is a 1 in 4 chance that a child born to parents who are carriers will have CF. In Scotland, there are about 750 people with CF, and 30 babies are born with CF every year. Carriers have no symptoms of CF and are often unaware of their status until genetically screened. CF affects several organs, especially the lungs and digestive system. The airways of the lungs in particular become coated in thick, sticky mucus: as a result, bacteria that are normally cleared are able to establish themselves in the airways, causing direct and indirect damage to the lungs. This leads to deterioration of lung function which over time is the major cause of early death in CF. Those born with CF today have a life expectancy of around forty years, thanks to the success of existing treatments which alleviate the symptoms of the disease. However, to make significant improvements to the quality of life and extend its duration, it is believed that new therapies targeting the root cause of the disease must be developed. Gene therapy is one such approach, and has the advantage that it would be applicable to anyone with CF. CF gene therapy research in Edinburgh is carried out by groups of scientists and clinicians in the Centre for Molecular Medicine and the Respiratory Unit at the Western General Hospital, the Royal Hospital for Sick Children, and the Roslin Institute at Easter Bush. Our primary goal is to translate scientific advances in gene therapy for CF into practical treatments for people with CF. We are currently embarking on a major Phase IIb trial of a non-viral gene therapy formulation in collaboration with colleagues in Imperial College, London, and the University of Oxford. Nearly half of the 120 participants in the trial will be from Scotland. The clinical centre for the Scottish arm of the trial is the Western General Hospital. In parallel, and in close collaboration with our colleagues in London and Oxford, we are developing a new virus-based delivery system for CF gene therapy. Our expectation is that the optimised formulation will be more efficient than the non-viral one currently in trial. Thank you for your interest in our research work. Every penny donated will be spent locally in Edinburgh to help us reach our goal of bringing effective gene therapy to the clinic.
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