Researchers have identified a new ALS gene: KIF5A.
WASHINGTON, March 21, 2018 /PRNewswire-USNewswire/ -- Researchers have identified a new ALS gene: KIF5A. This important gene discovery, funded by The The ALS Association through ALS Ice Bucket Challenge donations, will help researchers understand ALS disease pathways and represents a potential new ALS therapeutic target.
This was a large collaborative effort involving over 250 researchers, led by John Landers, Ph.D., Professor of Neurology at University of Massachusetts Medical School in Worcester, Mass., and Bryan Traynor, M.D., Ph.D., senior investigator in the Laboratory of Neurogenetics at the National Institute on Aging at the National Institutes of Health (NIH) in Bethesda, M.D. The discovery was published in the March 21 edition of the journal Neuron.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure.
Researchers from organizations Project MinE, Genomic Translation for ALS Care, Answer ALS, the Target ALS Postmortem Tissue Core, the New York Genome Center (NYGC) ALS Consortium, the CReATe Consortium, the NIH, and others came together to collaborate and share detailed genetic and clinical information needed to make this important discovery. The ALS Ice Bucket Challenge enabled The ALS Association to support these unique collaborative initiatives in precision medicine to accelerate the discovery of new treatments for ALS.
“This global, open-source collaboration has been the underlying goal for building these large initiatives,” said Lucie Bruijn, Ph.D. MBA, chief scientist for The ALS Association. “It is only through these kinds of partnerships that we can accelerate the pace of research towards new treatments for ALS.”
“It was truly a pleasure to see the collaborative effort of so many groups coming together to help with our goal of identifying a novel ALS gene,” said Dr. Landers. “It demonstrates that any single group may be able to make significant findings on its own but together we can accomplish boundless advances.”
“This collaborative discovery of KIF5A gives greater insight into the cellular mechanisms that lead to motor neuron degeneration and identifies important new targets for future gene therapies,” said Dr. Traynor.
Funding for Project MinE also came through The ALS Association Georgia Chapter, The ALS Association Greater New York Chapter, and New Amsterdam City Swim.
