Genetic cure for muscular dystrophy 'a step closer': Treatment for muscle-wasting disease is possible, scientists say
Steve Connor is the Science Editor of The Independent. He has won many awards for his journalism, including five-times winner of the prestigious British science writers’ award; the David Perlman Award of the American Geophysical Union; twice commended as specialist journalist of the year in the UK Press Awards; UK health journalist of the year and a special merit award of the European School of Oncology for his investigative journalism. He has a degree in zoology from the University of Oxford and has a special interest in genetics and medical science, human evolution and origins, climate change and the environment.
Thursday 19 August 1993
Scientists believe the animal experiments show that gene therapy - injecting healthy genes into patients - for muscular dystrophy is now more than a theoretical possibility, although researchers emphasise in today's issue of the journal Nature that more work needs to be done.
Duchenne muscular dystrophy affects about one in every 3,500 boys and is one of the most common genetic diseases with no cure. Females rarely develop the symptoms but can be carriers. The signs of muscle wasting first appear at about three years old. Children are often confined to wheelchairs by the age of 11 and develop severe respiratory and heart problems that frequently prove lethal by their mid-twenties.
Ever since the gene for muscular dystrophy was discovered in 1986, scientists have thought that gene therapy could provide a cure, but the problems of inserting such a large gene - it is 100 times the size of a typical gene - into all affected muscle cells seemed insurmountable.
Jeffrey Chamberlain, assistant professor of human genetics at the University of Michigan Medical School, United States, who led the research team, said the work demonstrated for the first time that a cure was possible: 'We have clearly shown that if you can get the gene into the muscle you can cure the disease.'
He warned that the method used to cure the mice could not be used on humans because genetic manipulation of the very early embryo was too dangerous. 'If you can find an effective way to get the gene into humans, and you can control it properly, then you may have a cure. In this study we found a way to control the gene once it's in the muscle; now we need to find a way to deliver it (to muscles).'
Dr Chamberlain's group is attempting to insert the healthy gene into the influenza virus which can then be used to 'infect' muscle cells of the lungs with the correct gene form.
Kay Davies, a medical geneticist who will soon become head of the Medical Research Council's new Clinical Research Centre at Hammersmith, London, and who helped to discover the muscular dystrophy gene, said at the International Congress of Genetics at Birmingham yesterday that Dr Chamberlain's work showed that such gene therapy does not produce toxic side-effects.
'It's a very important experiment (because) we need to know what range of toxicity there might be,' she said. 'This is a valuable contribution but we still have a long way to go before we get a method that is efficient at getting at all the respiratory cells.'
Although muscular dystrophy is inherited, about a third of cases result from spontaneous mutations in early life, so pre-natal diagnosis cannot eliminate it completely, which is why gene therapy is important.
Gods who sell our genes, page 32
- 1 The black and blue dress: Makers considering a white and gold version
- 2 Man to be beheaded in Saudi Arabia after ripping up a Koran and hitting it with his shoe
- 3 Husband and wife die holding hands within hours of each other after 67 years of marriage
- 4 PornHub turns masturbation into energy in bid to save the planet
- 5 The remarkable archaeological underwater discovery that could open up a new chapter in the study of European and British prehistory
Stephen Hawking's wife Jane Wilde on their marriage breakdown: 'The family were left behind'
PornHub turns masturbation into energy in bid to save the planet
Boko Haram: Thousands march in Cameroon to protest against the Islamist terror movement
White and gold or blue and black – what colour is the dress? An eyewitness gives a definitive answer
Kelly Osbourne leaves Fashion Police after Zendaya dreadlocks racism row
New theory could prove how life began and disprove God
This is what it's like to be dead, according to a guy who died for a bit
'Cash for access' scandal: Sir Malcolm Rifkind says 'unrealistic' for MPs to live on £67,000 salary
'Jihadi John': CAGE representative storms off Sky News accusing Kay Burley of Islamophobia
Ukip would cut billions from Scottish budget to fund English tax cuts
Russia's roadmap for annexing eastern Ukraine 'leaked from Vladimir Putin's office'
£9 per hour: Recruitment Genius: This role is based within a small family run ...
£28000 - £32000 per annum: Recruitment Genius: This Design and marketing agenc...
£46000 per annum: Recruitment Genius: This property investment firm are lookin...
£18000 - £35000 per annum: Recruitment Genius: This company specialises provid...