MDA awarded a grant totaling $412,500 over a period of three years to Eric Huang, professor of neuropathology at the University of California in San Francisco. The funds will help support Huang’s work to create cellular and mouse models of amyotrophic lateral sclerosis (ALS) that is caused by mutations in the fused in sarcoma (FUS) gene.
Genetic data have shown that mutations in the FUS gene can be identified in more than 5 percent of people with familial (inherited) ALS.
Huang and colleagues hypothesize that these FUS mutations interfere with the normal production of proteins, ultimately leading to the degeneration and death of the nerve cells called motor neurons.
The team’s goal is to create cellular and transgenic mouse models of FUS-related ALS that can be used to determine the ways in which mutant FUS proteins cause motor neuron degeneration.
Studies in the new FUS transgenic mice are expected to provide novel insights into the disease process and therapeutic targets for ALS, Huang said.