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 political parties aren't all the same – which means 2015 will be a 'big-choice' election
- 2 President of Argentina adopts Jewish godson to 'stop him turning into a werewolf'
- 3 ALS ice bucket challenge co-founder Corey Griffin drowns, aged 27
- 4 The 'Black Museum': After 150 years, public set to see exhibits from police’s grisly crime museum
President of Argentina adopts Jewish godson to 'stop him turning into a werewolf'
Exclusive: Abusers using spyware apps to monitor partners reaches 'epidemic proportions'
ALS ice bucket challenge co-founder Corey Griffin drowns, aged 27
UK weather: Warning for more snow and ice as freezing temperatures and gales hit Britain
New York police shooting: thousands turn out for the funeral of Rafael Ramos
British actor Idris Elba cannot star as James Bond because he is black, says shock jock Rush Limbaugh
Germany anti-Islam protests: 17,000 march on Dresden against 'Islamification of the West'
Ukip member gets into Christmas spirit with Union Flag plea to Santa 'for our country back'
Immigrants make UK racist, says Ukip councillor Trevor Shonk
BBC director Danny Cohen: Rising UK antisemitism makes me feel more uncomfortable than ever
Nigel Farage: Ukip leader named 'Briton of the year' by The Times
Not specified: Selby Jennings: VP/SVP Credit Quant Top tier investment bank i...
£40000 - £43000 per annum + benefits: Ashdown Group: Senior Marketing Executiv...
£40000 - £43000 per annum + benefits: Ashdown Group: An international organisa...
£25000 - £30000 per annum: Ashdown Group: Internal Recruiter -Rugby, Warwicksh...