Self Repair Gene Therapy Research
Gene therapy experts say they have found a way to persuade cells to repair themselves.
Instead of replacing a faulty gene, the new approach harnesses the cells' own correctional mechanisms.
Details of the research are being presented to the European Society of Human Genetics conference in Amsterdam.
This work shows that it may be possible to stimulate a related gene, and reduce the symptoms of the disease
The researchers, from the Institute of Genetics at the University of Cologne investigated a drug called valproate, which is already used to treat epilepsy.
The research focussed on spinal muscular atrophy (SMA), a relatively common inherited disease, and the leading cause of death in infants, affecting about one in every 6,000 newborns.
Lab tests had shown it could increase levels of the SMN protein by up to four times.
Professor Brunhilde Wirth said: "The long-term outcome could be both improved therapy to enable a better quality of life for SMA patients, and also the introduction of neonatal screening so that therapy could be started before the first symptoms appear."
Dr Fred Kavalier, of the British Society of Human Genetics, said: "This work shows that it may be possible to influence the behaviour of genes with drugs.
Source: Institute of Genetics, University of Cologne
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