Swiss scientists have made good progress toward treating spinal muscular atrophy (SMA), one of the leading genetic causes of early childhood death, the official Swissinfo news website reported Friday.
One out of 6,000 newborns is affected by the disease, which attacks nerve cells in the spinal cord responsible for voluntary movement.
Children suffering from the disease have two missing or malfunctioning genes that are needed for the production of a critical protein for healthy muscles.
According to the report, cellular biologist Daniel Schumperli and his team at Bern University have found ways to correct the problem by injecting cells with a specially developed gene, which helps synthesize the missing proteins.
The scientists were able to see significant improvements in mice with even the most severe cases of spinal muscular atrophy.
"This new study shows for the first time that the methods lead to a notable reduction in disease symptoms," Schumperli said.
The results have been published in the journal Human Molecular Genetics.
Source:Xinhua
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