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Gene therapy in the news

23 September 2002
By BioNews
Appeared in BioNews 176

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 the muscles.

The researchers, from the Seattle base of the University of Washington, US, injected a modified cold virus carrying the gene into the hind legs of mice with a condition similar to Duchenne muscular dystrophy. After a month, the mice were seen to have a 40 per cent improvement in muscle action. The research is published in the Proceedings of the National Academy of Sciences.

In addition, researchers in Finland have found that gene therapy may help to prevent arteries becoming blocked again after surgical procedures to clear them. When angioplasty - the insertion of a balloon-like catheter into an artery to unblock it - is performed, a metal tube called a stent is sometimes used to prop the artery open. This can trigger a reaction in some patients which causes the artery to rapidly re-block.

Now, the researchers, from the University of Kuoppio in Finland, believe they have discovered why this happens. They believe the stent can damage the lining of the artery walls, triggering the rapid growth of new cells which contribute to the re-blocking of the artery. Damage also causes 'distress signals' to be sent out, which cause the body to produce chemicals called 'free radicals', which in turn cause more damage to the lining. They also believe the free radicals can be counteracted by the insertion of an extra gene which will cause another chemical to be produced, but which is not produced naturally by patients. The theory was tested in a rabbit model, using a modified virus to deliver the gene. After two and four weeks, it was observed that fewer free radicals were present in the rabbits' arteries.

Gene swap 'reverses' muscular dystrophy
BBC News Online |  16 September 2002
Gene therapy aims to fight artery re-clogging
Yahoo Daily News |  16 September 2002
Gene therapy keeps blood flowing
BBC News Online |  22 September 2002
Gene therapy may improve muscles
Yahoo Daily News |  16 September 2002
3 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...
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...
26 July 2004 - by BioNews 
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...
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