In New Research, Wright Finds ALS Gene Therapy Ineffective

By Christopher Sarachilli | June 18, 2015

Dr. Megan C. Wright, assistant professor of biology, and colleagues published new findings on Lou Gehrig’s disease, or amyotrophic lateral sclerosis (ALS), in Pain and Central Nervous System Week.

ALS causes both cellular abnormalities and rapid degeneration of motor neurons. These abnormalities cause irregular transportation of amino acids within the spinal cord, which in turn causes the degeneration of motor neurons. This usually leads to paralysis of the respiratory system and, eventually, death.

Wright and other researchers tested a specific gene therapy by injecting the spinal cords of mice with delivery viruses. The gene therapy spread rapidly and elevated levels of missing amino acid transporters, but did not stop the eventual respiratory paralysis, improve motor function, or prolong the animals’ survival. In this way, researchers discovered that the therapy was not effective in treating ALS.