TARDBP and amyotrophic lateral sclerosis: A heterozygous missense mutation, 1127G>A in the Exon 6 of the TARDBP gene has been discovered in an Italian family with several members affected by ALS.23 To try to put the bases for treatment, we identified a siRNA complementary to the region comprising the point mutation c.1127G>A that specifically targets the TDP-43 mutant allele and that can counteract the cellular phenotypes induced by mutant allele expression.