CF gene therapy in the news

Some of you may have seen the story in the news about research into using a 'super' virus in gene therapy for CF.

Here's our response to this story.

A spokesperson from the UK Cystic Fibrosis Gene Therapy Consortium said

“The techniques used in this research are innovative, but more work is needed before this could be translated into clinical benefit for people with CF. The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene; the long-term treatment of a CF patient would require repeat administration of the virus which is usually not feasible as the body develops immune responses against the virus; and although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient.

The UK CF Gene Therapy Consortium funded by the Cystic Fibrosis Trust is currently trialling gene therapy in patients for clinical benefit. Our Wave 1 gene therapy research is using a liposome to get the gene therapy product into the body. Following this year's pilot study in humans, we anticipate going to a major multi-dose clinical trial in 2010.”