The identification and isolation of the CF gene in 1989 meant that scientists could look to find ways of correcting the basic CF genetic defect rather than just treating the symptoms of the condition. The best way to do this is through gene therapy – adding normal copies of the faulty gene.
As lung damage is the major cause of illness and death in CF, the first attempts at gene therapy are focusing on delivering a healthy copy of the CF gene to the lungs.
UK based researchers have already demonstrated proof-of-principle for correction of the genetic defect in the human CF nose and lung. The next step is to develop this principle into an effective treatment for people with Cystic Fibrosis. This won’t be an easy process; gene therapy is still in its infancy and there are many problems to overcome.
The Cystic Fibrosis Trust has brought together the UK’s leading CF researchers to form the UK CF Gene Therapy Consortium. The scientists are based in Edinburgh, London and Oxford.