These data further indicate future research directions, suggesting that it will be necessary to identify target molecules, including nuclear proteins and/or RNA species that associate with FUS, in order to elucidate the molecular mechanisms leading to neuronal dysfunction in FUS-associated ALS/FTLD and to develop the disease-modifying therapies that are eagerly desired in those relentless neurodegenerative diseases. The gene discussed is FUS; the disease is neurodegenerative disease.