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King's College London - Health: More than a medical matter

Improved gene therapy for muscles

26 July 2004

By BioNews

Appeared in BioNews 268

Researchers have overcome one of the major hurdles in using gene therapy to treat muscle diseases: delivery of the gene to muscles throughout the body. The study, to be published in the journal Nature Medicine next month, is further progress towards treating diseases such as Duchenne muscular dystrophy (DMD), which is the most common lethal childhood genetic disease.

Muscular dystrophy is the generic name for a group of diseases that are caused by faults in a gene known as dystrophin. Gene therapy has previously been used to treat muscular dystrophies, by injecting viruses carrying the gene directly into muscle, but the latest research uses the bloodstream to deliver the gene to muscles throughout the body.

The research team, based at the University of Washington (US), conducted experiments on mice lacking the dystrophin gene, which exhibit similar symptoms to DMD sufferers. The team were able to reverse these symptoms by delivering a copy of the gene using a particular type of virus. This virus, called adeno-associated virus (AAV) pseudotype 6, is able to target muscle cells, so the gene is only delivered to those cells that need it. As the virus is unable to replicate on its own, it poses little risk to humans.

The naturally occurring form of the dystrophin gene is too large to fit inside a virus, so the researchers had to use a scaled down version, known as micro-dystrophin. In a further use of clever science, the team combined the gene therapy with drugs that increased the permeability of the blood vessels, allowing the viruses to leave the bloodstream and deliver the gene to the muscle.

'By giving one single injection of this AAV carrying a dystrophin gene into the bloodstream, we are able to deliver therapeutic levels of dystrophin to every skeletal and cardiac muscle of an adult, dystrophic mouse', said Professor Jeffery Chamberlain, leader of the research team. Chamberlain, director of the University of Washington's Muscular Dystrophy Co-operative Research Centre, is currently putting together a proposal for a trial of this procedure in humans. Despite saying that further work on animals was needed, he expressed hopes of full-scale human trials in five years with widespread use in ten.

Although developed with muscular dystrophy in mind, the technique should allow delivery of a wide variety of genes to the muscle. This means it has wide-ranging potential to treat muscular ailments, but raises further questions about the use of genes for cheating in sport. Chamberlain shrugs off these fears saying, 'it would be really easy to detect because the adeno-associated virus would stay around for years in the body'.


Gene therapy promises cure for muscular dystrophy
The Independent | 26 July 2004
Medical News Today | 26 July 2004
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
Nature Medicine | 25 July 2004


03 April 2006 - by BioNews 
A team of scientists based in Frankfurt, Germany, has become the world's first to treat a rare and 'incurable' hereditary immune disease in adults, using gene therapy. Gene therapy has been used successfully in the past, but this has generally been in the treatment of children. Over the past two... [Read More]
30 March 2006 - by BioNews 
US researchers have begun a clinical trial to test a new gene therapy treatment for Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder. The team, based at the Columbus Children's Hospital in Ohio, will test the safety and effectiveness of gene injections in six affected boys aged 8-12 years... [Read More]

29 March 2004 - by BioNews 
Gene therapy trials for haemophilia, muscular dystrophy and childhood blindness are to receive £3 million funding, the UK Department of Health has announced. A further £1 million has been set aside for research into the long-term safety of some gene therapy techniques. The funding is part of a £50 million... [Read More]
18 February 2004 - by BioNews 
Rats injected with a gene that promotes tissue growth develop stronger muscles after regular exercise, according to research presented at the annual meeting of the American Association for the Advancement of Science (AAAS) in Seattle. Scientists based at the University of Pennsylvania in Philadelphia, US, are hoping to develop new... [Read More]
14 July 2003 - by BioNews 
Two new studies promise new treatments for the muscular dystrophies, a group of genetic conditions that cause progressive muscle weakness. Italian researchers have combined stem cell and gene therapy techniques to successfully treat mice with symptoms of limb-girdle muscular dystrophy, while a UK group has used a novel form of... [Read More]
23 September 2002 - by BioNews 
Gene therapy has been used for the first time to relieve some of the effects of muscular dystrophy. The effects of the condition, a severe inherited muscle-wasting disease, were partially corrected in mice when a new gene was introduced that restored levels of dystrophin, a protein lacked by sufferers, to... [Read More]
04 December 2000 - by BioNews 
A team of US scientists has developed a new gene therapy treatment for Duchenne muscular dystrophy (DMD), a severe inherited muscle-wasting disease. The researchers successfully treated laboratory mice with the condition, stopping and even reversing its symptoms. The study, carried out at the University of Pittsburgh, was published in the... [Read More]

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