Question: Have there been any successful gene therapies

Tim Craggs answered on 25 Jun 2010:

Hi Rooshq,

Gene therapies are in the early stages of research right now, but there are encouraging results for cystic fibrosis. This is taken form the cystic fibrosis website: http://www.cftrust.org.uk/

What is gene therapy?
The identification and isolation of the CF gene in 1989 meant that scientists could 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 – replacing faulty genes with normal ones.

As lung damage is the major cause of illness and death in CF, the first attempts at gene therapy are focusing on delivering genes to the lungs. UK based researchers have already demonstrated correction of the genetic defect in mice, the human CF nose and, most recently, in the human CF lung. The next step is to try to develop effective treatment for people with CF.

UK CF Gene Therapy Consortium
In around 2002, the Cystic Fibrosis Trust brought together the UK’s leading CF geneticists to form the UK CF Gene Therapy Consortium. The scientists are based in Edinburgh, London and Oxford. The Consortium now comprises 80 dedicated scientists and clinicians.

The scientists have developed a lead gene therapy product, which comprises the healthy copy of the gene and a carrier or vector to get it to the right place in the cells lining the airways. This has taken many years of hard, long and expensive work. For example, the healthy gene is in its 169th version.