Volunteers test a gene therapy for cystic fibrosis: Children would benefit most from genetic engineering technique that could be used to treat Britain's most common inherited disease

THE FIRST British attempts to use genetic engineering to treat the life-threatening disease cystic fibrosis (CF) were announced by doctors and scientists at the National Heart and Lung Hospital in London yesterday.

Over the next few weeks, nine volunteers at the hospital will have normal genes sprayed into the cells lining their noses and lungs, to counter the effects of the faulty genes that they have inherited.

If successful, the new therapy could be offered routinely to CF patients within five years, according to Dr Duncan Geddes, director of respiratory medicine at the hospital.

Children would benefit most, he said, if they got the treatment early enough, before lung damage occurs. 'If we can give paediatricians evidence that gene therapy is safe and that it works, then they see no reason not to give it to children.'

But Dr Geddes cautioned that it would be wrong to talk in terms of a 'cure', because the gene transplants will have to be given repeatedly.

Cystic fibrosis is the most common inherited disease in Britain, affecting more than 7,000 children and young adults. Their lungs clog with thick sticky mucus which provides a perfect breeding ground for bacteria and leaves them open to recurrent infections. These scar the lungs and eventually lead to lung failure.

There is no cure, and people with CF used to die in early childhood. With modern antibiotics and intensive nursing, people with CF now have a much longer life expectancy.

In 1989, after more than a decade of effort by scientists around the world, researchers at the universities of Michigan, in the United States, and Toronto, in Canada, isolated the gene responsible for the disease. The defective CF gene fails to control the passage of salt and water in and out of the body's cells. In the lungs, this failure results in the sticky mucus. By replacing faulty CF genes with normal ones the defect can be corrected.

However, the US trials, which began in April, have hit a problem and have been stopped. The doctors used a genetically engineered version of the common cold virus to infect the lungs of CF sufferers: the virus enters the cells lining the lung and thus transfers the normal CF gene into them. But the procedure caused inflammation in the lungs of some patients, who were already sensitive to infections.

In contrast, Professor Bob Williamson of St Mary's Hospital Medical School believes that the technique he has devised for the British trials may be milder and less liable to side effects. Instead of employing a virus, he has found a way of using fat globules, called liposomes, to carry the genes. Cells have a fatty outer membrane, Professor Williamson explained yesterday, and the two will join just as 'two oil drops floating on water will fuse and form a single drop'.

The clinical trials announced yesterday are the first for CF to be held outside the US. The safety and efficacy of the procedure have been extensively tested, partly on mice with a defect analogous to CF, and the ethics have been examined by a government committee on gene therapy, under the chairmanship of Sir Cecil Clothier.

One concern was that, because no one can know the full consequences, new genes should not be passed down to future generations, and it was decided that initially only men should take part, since virtually all men with CF are infertile.

(Graphic omitted)

News
The cartoon produced by Bruce MacKinnon for the Halifax Chronicle-Herald on Thursday, showing the bronze soldiers of the war memorial in Ottawa welcoming Corporal Cirillo into their midst
news
News
The Edge and his wife, Morleigh Steinberg, at the Academy Awards in 2014
peopleGuitarist faces protests over plan to build mansions in Malibu
Voices
Nigel Farage has backed DJ Mike Read's new Ukip song
voicesNigel Farage: Where is the Left’s outrage over the sexual abuse of girls in the North of England?
Life and Style
The Zinger Double Down King, which is a bun-less burger released in Korea
food + drinkKFC unveils breadless meat beast
PROMOTED VIDEO
Have you tried new the Independent Digital Edition apps?
News
ebooksAn unforgettable anthology of contemporary reportage
Latest stories from i100
Have you tried new the Independent Digital Edition apps?
Independent Dating
and  

By clicking 'Search' you
are agreeing to our
Terms of Use.

iJobs Job Widget
iJobs General

SSRS Report Developer - Urgent Contract - London - £300pd

£300 Per Annum: Clearwater People Solutions Ltd: SSRS Report Developer – 3 Mon...

KS1 Teacher

£95 - £150 per day: Randstad Education Birmingham: Key Stage 1 teacher require...

HR Business Partner - Essex - £39,000 plus benefits

£32000 - £39000 per annum + benefits + bonus: Ashdown Group: Generalist HR Man...

Trainee Recruitment Consultant

£18000 - £30000 per annum + uncapped: SThree: Do you feel like your sales role...

Day In a Page

How could three tourists have been battered within an inch of their lives by a burglar in a plush London hotel?

A crime that reveals London's dark heart

How could three tourists have been battered within an inch of their lives by a burglar in a plush London hotel?
Meet 'Porridge' and 'Vampire': Chinese state TV is offering advice for citizens picking a Western moniker

Lost in translation: Western monikers

Chinese state TV is offering advice for citizens picking a Western moniker. Simon Usborne, who met a 'Porridge' and a 'Vampire' while in China, can see the problem
Handy hacks that make life easier: New book reveals how to rid your inbox of spam, protect your passwords and amplify your iPhone

Handy hacks that make life easier

New book reveals how to rid your email inbox of spam, protect your passwords and amplify your iPhone with a loo-roll
KidZania lets children try their hands at being a firefighter, doctor or factory worker for the day

KidZania: It's a small world

The new 'educational entertainment experience' in London's Shepherd's Bush will allow children to try out the jobs that are usually undertaken by adults, including firefighter, doctor or factory worker
Renée Zellweger's real crime has been to age in an industry that prizes women's youth over humanity

'Renée Zellweger's real crime was to age'

The actress's altered appearance raised eyebrows at Elle's Women in Hollywood awards on Monday
From Cinderella to The Jungle Book, Disney plans live-action remakes of animated classics

Disney plans live-action remakes of animated classics

From Cinderella to The Jungle Book, Patrick Grafton-Green wonders if they can ever recapture the old magic
Thousands of teenagers to visit battlefields of the First World War in new Government scheme

Pupils to visit First World War battlefields

A new Government scheme aims to bring the the horrors of the conflict to life over the next five years
The 10 best smartphone accessories

Make the most of your mobile: 10 best smartphone accessories

Try these add-ons for everything from secret charging to making sure you never lose your keys again
Mario Balotelli substituted at half-time against Real Madrid: Was this shirt swapping the real reason?

Liverpool v Real Madrid

Mario Balotelli substituted at half-time. Was shirt swapping the real reason?
West Indies tour of India: Hurricane set to sweep Windies into the shadows

Hurricane set to sweep Windies into the shadows

Decision to pull out of India tour leaves the WICB fighting for its existence with an off-field storm building
Indiana serial killer? Man arrested for murdering teenage prostitute confesses to six other murders - and police fear there could be many more

A new American serial killer?

Police fear man arrested for murder of teen prostitute could be responsible for killing spree dating back 20 years
Sweetie, the fake 10-year-old girl designed to catch online predators, claims her first scalp

Sting to trap paedophiles may not carry weight in UK courts

Computer image of ‘Sweetie’ represented entrapment, experts say
Fukushima nuclear crisis: Evacuees still stuck in cramped emergency housing three years on - and may never return home

Return to Fukushima – a land they will never call home again

Evacuees still stuck in cramped emergency housing three years on from nuclear disaster
Wildlife Photographer of the Year: Intimate image of resting lions claims top prize

Wildlife Photographer of the Year

Intimate image of resting lions claims top prize
Online petitions: Sign here to change the world

Want to change the world? Just sign here

The proliferation of online petitions allows us to register our protests at the touch of a button. But do they change anything?