Gene Therapy for Cystic Fibrosis | Redbrick | University of Birmingham

Gene Therapy for Cystic Fibrosis

With gene therapy for cystic fibrosis on its way, the outlook for sufferers is looking better than ever. Sci&Tech editor, Rachel Taylor reports.

Cystic fibrosis (cf) is one of the world’s most common genetic diseases. A sufferer’s lungs and digestive system become congested with thick, sticky mucus, meaning that they suffer with breathing difficulties and weight gain. Every new-born child in the UK is screened for the disease with a blood spot test. Around 1 in 2,500 babies born in the UK will have cystic fibrosis, with the condition more prominent in the Caucasian population, especially those of Northern European descent.

sufferers born in this century are expected to live past 50
Today there are over 9,000 people living with the disease, with those born in this century expected to live past 50. A few decades ago the outlook for cf sufferers was not so positive, most would die of related complications before adulthood. However, now this has completely turned around and not only are people expected to live past 50, but with a new treatment from the UK Cystic Fibrosis Gene Therapy consortium, they may go on to have a much longer, healthier life expectancy.

In 1985 when the cystic fibrosis-causing gene was discovered on chromosome 7, it was the single most important discovery in cystic fibrosis research, and this year it has finally paved the way for successful gene therapy!

Blausen_0286_CysticFibrosisAlthough the improvement of 3.7% may seem small, this will continue to improve over time and an improvement means the world to a sufferer. It also gives hope to researchers that after 30 years they can replace the faulty cf gene with a healthy one. However, it may be a long way off until we see drastic improvements. Deborah Gill, whose team were part of the consortium explains that the therapy “isn't ready to prescribe to patients, personally I am delighted with the result – it's better than I expected” and that the “possibility of being able to increase the dose to see a greater effect in more patients is very exciting." The lungs of a cf sufferer generally decrease in function by around 3% each year, so the treatment could definitely have the impact to prevent further damage.

Gene therapy has often been associated with Cystic fibrosis as it is very common and only caused by a single gene. Finding a way of getting a treatment into well-protected lung tissue, however, has been one of the major difficulties. There were 116 people in the trial, half getting 9 monthly doses of gene therapy and the other half observed as a control.

There is hope that now the therapy for cystic fibrosis has been unlocked it may pave the way for treating other similar genetic diseases.

Sci & Tech Editor. Biological Sciences student. Keenly interested in the subjects of plant sciences and genetics. (@Rachel_Taylor95)


9th July 2015 at 12:00 pm

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US Air Force and Bruceblaus