Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease primarily affecting motor neurons. Recently,1 Miller et al. presented phase 3 data for the RNase-H-recruiting antisense oligonucleotide (ASO) targeting superoxide dismutase 1 (SOD1) in ALS, which represents a step forward in the deployment of ASO therapeutics against CNS targets.
Med (N Y)
733 - 734
Humans, Amyotrophic Lateral Sclerosis, Ribonuclease H, Neurodegenerative Diseases, Superoxide Dismutase-1, Superoxide Dismutase, Oligonucleotides, Antisense