Targets were selected on the basis of direct links to ALS/FTD or other neurological diseases (e.g. ATXN2, KIF1C, PEX19, ARRB2), or because of documented involvement in pathways implicated in ALS/FTD such as excitotoxictiy (GABBR1, CABIN1), protein degradation (PSMD4, BAT3), cytoskeletal remodeling and neuritogenesis (PPP1R12C) and RNA metabolism (PAN2, RNH1, DAZAP1). The gene discussed is GABBR1; the disease is amyotrophic lateral sclerosis.