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New genetically engineered mice aid understanding of incurable neuromuscular disease, say University of Missouri researchers

Apr 17, 2012

This story gets at the heart of the One Health/One Medicine initiative – using an animal model to test treatments for human disorders and diseases.

From MU News Bureau

COLUMBIA, Mo. — A team of scientists from the University of Missouri created a genetically modified mouse that mimics key features of Charcot-Marie-Tooth disease, an inherited neuromuscular disease affecting approximately 150,000 people in the United States.

Charcot-Marie-Tooth, or CMT, is a group of progressive disorders that affects the peripheral nervous system, the part of the nervous system that connects the brain and spinal cord to targets such as muscles. The disease largely affects the distal nerves, those running to the feet and hands, and can progress to include the legs and arms.

“Wasting and weakening of the muscles occurs because the distal nerves are either dying or not functioning properly,” said Michael Garcia, study leader and associate professor of biological sciences. “The condition can be very debilitating depending on the muscles affected and the degree to which they are affected.”

No cure exists for CMT, but Garcia hopes that insights gleaned from the new mouse model may aid the development of therapeutic interventions. More