Novel Gene Therapy to treat Cystic Fibrosis

Winner of The Medical Futures Innovation Award - MRC Translational Research Innovation Award 2011
Cystic Fibrosis Gene Therapy
Best Therapeutic Innovation in the Respiratory Innovation Awards
Professor Eric Alton, Dr Chris Boyd, Dr Jane Davies, Dr Deborah Gill, Dr Uta Griesenbach, Ms Tracy Higgins, Dr Stephen Hyde, Dr Alastair Innes and Professor David Porteous.
Imperial College, London, University of Edinburgh & University of Oxford
What is it?
This will be a new medical treatment for cystic fibrosis, a disease which clogs up the lungs and digestive tract with thickened secretions causing difficulty breathing and digestion of food, with progressive disability and often early death.
What’s new?
The innovation is a novel ‘friendly’ virus that delivers a normal version of the faulty cystic fibrosis gene into the lungs of patients. One dose generates large amounts of the normal protein; an effect sustained for at least two years in animal models, and can be given repeatedly for life-long therapy.
Who will use it?
Cystic Fibrosis affects more than 70,000 people worldwide. Only half of those with the disease are expected to live past their mid 30‘s. In the UK alone, more than 2 million people carry the faulty gene that causes Cystic Fibrosis.
The cost of care for a patient varies between £20,000 to £100,000 per year with existing treatments.
@Edinburghuni @Uniofoxford @imperialcollege @the_mrc
#innovation #healthcare #nhs #bbc #bbcnews #medicaldevice #money #entrepreneurs #change #future #revolutionary#lungdisease #genetherapy #cysticfibrosis #universityofoxford #universityofedinburgh #imperialcollege #themrc #translationalresearch
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