University of Missouri researchers have found that synthetic molecules might prove to be a key in reducing the severity of Spinal Muscular Atrophy, or SMA, the leading genetic cause of infantile death.

The work is led by Chris Lorson, a researcher at the Bond Life Sciences Center and a professor in both the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology.

SMA is a genetic disease inherited by one in 6,000 children who are missing a gene, SMN-1, that produces a protein needed to direct spinal nerves to command muscles. Humans have a partially functioning backup gene, SMN-2, that makes a small amount of the needed protein.

Lorson’s team introduced synthetic ribonucleic acid — a biologically fundamental molecular similar to DNA — into mice carrying genes responsible for the disease. Doing so, they found, triggered the backup gene and lowered the severity of SMA.

“The mice that receive synthetic RNA gain more weight, live longer and had improvements in motor skills,” Lorson said in a statement. “These results are very exciting.”

More work is needed before synthetic RNA can be used on humans, but Lorson said the results are promising.

He pointed out that there are similar trials being performed with the backup genes on other diseases, such as ALS, or Lou Gehrig’s disease.

“It’s been remarkable to watch how quickly SMN-2 knowledge has transformed from basic molecular biology to being modified targets for novel therapeutics,” he said. “SMN-2 is like a light that’s been dimmed, and we’re trying anything to get it brighter. Even turning it up a little bit would help dramatically.”

Lorson’s study, which he co-authored with Erkan Osman and Pei-Fen, was published in the journal Molecular Therapy.

© 2011 The Columbia Daily Tribune