A team of University of Missouri researchers have found that targeting a synthetic molecule to a specific gene could help the severity of the disease Spinal Muscular Atrophy (SMA).
“When we introduced synthetic RNA into mice that carry the genes responsible for SMA, the disease’s severity was significantly lowered,” said Chris Lorson, researcher at the Bond Life Sciences Center and professor in the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology. “The mice that receive synthetic RNA gain more weight, live longer, and had improvements in motor skills. These results are very exciting.”
While the results are promising, Lorson notes additional research is needed before synthetic RNA could be used on humans for SMA. Clinical trials for similar synthetic RNAs are currently being performed in other neurodegenerative disease such as Lou Gehrig’s or ALS.
“It’s been remarkable to watch how quickly SMN-2 knowledge has transformed from basic molecular biology to being modified targets for novel therapeutics,” Lorson 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.”
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