Protein target found for ALS
This study, which received support from The ALS Association, shows that SUPT4H1 could be a potential target for researchers as they develop therapy for people with ALS that is from C9orf72 genetic expansion.
“This study highlights a potentially important new target for therapy development,” Dr. Lucie Bruijn, chief scientist for The ALS Association, said. “Current efforts to reduce expression of the expanded C9orf72 gene with antisense therapy will continue, and we are very excited about the prospects of this approach. At the same time, every effort must be made to develop other promising approaches -- and these results suggest that targeting SUPT4H1 may offer a new and powerful route to treatment of ALS due to this gene mutation.”
ALS, a progressive neurodegenerative disease, damages the nerve cells located in the spinal cord and the brain. People who are diagnosed with ALS gradually lose all muscle control and movement, becoming completely paralyzed. Within two to five years of receiving their diagnosis, most ALS patients die. Scientists have not yet learned why veterans are two times more likely to have ALS than the civilian population.
Organizations in this story
ALS Association 1275 K Street Northwest Washington, DC - 20005