2/28/2010 PERMALINK
New gene therapy reverses effects of lethal muscle disorder in mice. Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for a lethal childhood muscle-wasting disease where not enough survival motor neuron protein, or SMN is produced. This reduced protein in motor neurons specifically is caused by the absence of a single gene. The researchers used an altered virus to deliver a portion of DNA that makes the SMN protein into the veins of newborn mice ranging in age from 1 to 10 days old. The SMN-laced viral vector injected into the youngest mice reached almost 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 at Ohio State University reported.