COLUMBUS, Ohio, March 2 (UPI) -- U.S. scientists say gene therapy can correct motor function, restore nerve signals and improve survival in mice with childhood spinal muscular atrophy.
Ohio State University researchers said they used gene therapy to reverse a protein deficiency as a treatment for the muscle-wasting disease that results when a child's motor neurons produce insufficient amounts of what is called survival motor neuron protein, or SMN. That reduced protein in motor neurons is caused by the absence of a single gene.
The OSU scientists said they used an altered virus to deliver a portion of DNA that makes the SMN protein into the veins of newborn mice. The SMN-laced viral vector injected into the youngest mice reached nearly half of their motor neurons, resulting in improved muscle coordination, properly working electrical signals to the muscles and longer survival than in untreated mice, scientists said.
"We're replacing what we know is lost," said Professor Arthur Burghes, a senior study co-author. "And we have shown that when you put the protein in postnatally, it will rescue the genetic defect.
"This technique corrects the mice considerably more than any drug cocktails being studied as a potential treatment in humans," he added.
The researchers -- who say they hope to progress to human clinical trials as soon as required toxicology experiments are completed -- report their findings in the online early edition of the journal Nature Biotechnology.
