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Researchers Develop New Drug For Spinal Muscular Atrophy

Update Date: Sep 16, 2014 03:46 AM EDT

Nearly one out of every 40 individuals in the United States carries the genes responsible for spinal muscular atrophy (SMA) - a neurodegenerative disease that causes muscles to weaken over time. Now, researchers have achieved a breakthrough with the development of a new compound found to be highly effective in animal models of the SMA. 

"The strategy our lab is using to fight SMA is to 'repress the repressor,'" said Chris Lorson, a researcher in the Bond Life Sciences Center and professor in the MU Department of Veterinary Pathobiology, in the press release. "It's a lot like reading a book, but in this case, the final chapter of the book-or the final exon of the genetic sequence-is omitted. The exciting part is that the important chapter is still there-and can be tricked into being read correctly, if you know how. The new SMA therapeutic compound, an antisense oligonucleotide, repairs expression of the gene affected by the disease."

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

Muscles in the lower extremities are usually affected first, followed by muscles in the upper extremities, including areas around the neck and spine, the press release added.

The research had found that the earlier the treatment can be administered in mice with SMA, the better the outcome.

The study was carried out at University of Missouri-Columbia. 

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