ISU researchers find possible treatment for spinal muscul...(work is that we have this stuff called j...)

The resulting proteins are normal just like a regular cell - free from Spinal Muscular Atrophy.

"Our cells are healthy and survive," he said. "From that point of view, this is a major achievement."

Singh, along with his team Natalia Singh and Maria Shishimorova, both of Iowa State University's biomedical services department; Lu Cheng Cao, University of Massachusetts Medical School, Worcester; and Laxman Gangwani, Medical College of Georgia, Augusta, have their research highlighted as the cover story on this month's issue of the journal RNA Biology. Their research (link to research) is the most downloaded story on the RNA Biology page of the Web site Landes Bioscience.

Spinal Muscular Atrophy affects 1 in 6,000 to 1 in 10,000 children born every year. One in 40 people are carriers of the disease -- they don't have the symptoms, but could pass the disease to their children.

Most children born with the most severe type of SMA die within two years.

Using this junk sequence in SMN2 to restore the high levels of functional SMN protein could eliminate Spinal Muscular Atrophy caused by deletion or mutation in SMN1.

Singh believes this technology could also work treating other diseases.

"We know that Parkinson's disease, Alzheimer's disease, cystic fibrosis, multiple sclerosis and cancer all come from genes that are aberrantly spliced," he said.

"If this is a model disease, meaning we succeed in treating Spinal Muscular Atrophy, we will know how to correct splicing of other genes in other diseases," he said.

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