ALS – Jeffrey Rothstein, M.D., Ph.D.

ALS - Jeffrey Rothstein, M.D., Ph.D.
“This research project will provide a fundamental understanding of what causes the most common form of ALS — that is, the actual ‘tipping point’ of what initiates disease injury,” Jeffrey Rothstein says. “This will provide the best understanding and tools to someday stop the disease.”

Jeffrey Rothstein, professor of neurology and neuroscience at Johns Hopkins University School of Medicine in Baltimore, was awarded an MDA research grant totaling $300,000 over three years to elucidate the mechanisms underlying ALS (amyotrophic lateral sclerosis) caused by mutations in the C9ORF72 gene. Rothstein will use ALS patient-derived stem cells to investigate the role of nuclear dysfunction in ALS caused by C9ORF72 mutations. The work could uncover novel drug targets for ALS.

Funding for this MDA research grant began Aug. 1, 2015.