Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by
motor neuron loss. ALS is now associated with mutations in numerous genes, many of which
cause disease in part through toxic gain-of-function mechanisms. Antisense
oligonucleotides (ASOs) are small sequences of DNA that can reduce expression of a target
gene at the post-transcriptional level, making them attractive for neutralizing mutant or toxic
gene products. Advancements in the medicinal chemistries of ASOs have improved their …