Gene therapy to treat cystic fibrosis

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The Independent Online
WITHIN five years human gene therapy will become a routine treatment for cystic fibrosis - the most common inherited disease in Britain - says one of the UK's pioneers of human gene therapy.

However, Professor Bob Williamson, professor of Molecular Genetics at St Mary's Hospital Medical School, in London, said that neither of the techniques currently in clinical trials was likely to be adopted directly. He was notably cautious about the success of trials for introducing intact genes into the lungs of cystic fibrosis patients.

Cystic fibrosis is a chronic debilitating disease in which the lungs clog with mucus. Although repeated infections can be controlled by antibiotics, few CF sufferers survive much beyond 30.

Researchers have two methods of introducing the intact genes to patients' lungs. The approach adopted in the US is to use a genetically engineered adenovirus, similar to the virus that causes the common cold, to infect the cells lining the lungs with the new gene. In Britain researchers have sprayed liposomes - fatty particles resembling cell membranes - into the noses of patients to try to inflitrate the genes into cells lining the airway.

Professor Williamson said the adenovirus technique was very efficient but provokes inflammation and an immune response. The liposome data indicates safety but is less efficient. 'My view is that at the end of the day we will all see something that is neither liposome nor adenovirus,' he said.

Seven of Britain's leading geneticists yesterday condemned the way decisions on the safety of genetic engineering have been shrouded in secrecy. The government committees which decide whether or not to approve human gene therapy and whether it is safe to release genetically engineered plants, viruses and bacteria into the environment ought to be open to public scrutiny, they said.

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