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Researcher develops new spinal muscular atrophy treatment

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A researcher at the University of Missouri has developed a new compound found to be highly effective in animal models of spinal muscular atrophy, according to a recent press release.

Approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time, according to the press release.

In individuals with SMA, the spinal motor neuron-1 (SMN1) gene is mutated and lacks the ability to process a key protein that helps muscle neurons function.

However, humans have a nearly identical copy gene, SMN2, and Chris Lorson, PhD, a researcher in the Bond Life Sciences Center and professor in the department of veterinary pathobiology at the University of Missouri, has developed a drug that targets that specific genetic sequence to allow proper editing of the SMN2 gene.

The drug allows the SMN2 gene to bypass the defective gene and process the protein that helps the muscle neurons function.

Lorson patented the compound in April. The drug improved survival rate by 500% to 700% in mice studies, with a 90% improvement observed in severe SMA cases, according to the press release.

Reference: www.missouri.edu