Ben-Gurion University researchers highlight gene’s potential in ALS therapy


( A new study published by researchers from the Ben-Gurion University of the Negev (BGU) describes a new molecular mechanism that could help develop new treatments for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease.

Ben-Gurion University of the Negev. Credit: Wikimedia Commons.

ALS is a fatal neurodegenerative disease that destroys motor neurons which control voluntary movement of the muscles. In 20 percent of genetic ALS cases, the disease is caused by a gene mutation that leads to the accumulation of misfolded proteins, which causes the death of motor neurons.

Published online in Proceedings of the National Academy of Sciences of the United States of America (PNAS), the study looked at mice and found that a gene regulating cell inflammation and immunity acts as a chaperone to the misfolded protein. When the researchers eliminated this gene, they found the accumulation of the protein increased and the disease accelerated.
"This study provides insight into the potential therapeutic role” of the studied gene, said Dr. Adrian Israelson, head of the Cellular and Molecular Neurodegeneration Lab in the BGU Department of Physiology and Cell Biology.

"Correct protein folding is critically important, which is why we are focusing on the diverse set of complex cellular mechanisms, including molecular chaperones, that promote efficient folding and prevent toxicity," Israelson said.

Posted on September 14, 2016 .