An RNA Nexus in Neurodegeneration

The causes of neuronal death in neurodegenerative diseases are multifaceted, but an emerging theme in a number of disorders is the role of RNA regulation and dynamics. In amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, RNAs arising from an expanded GGGGCC repeat are thought central to the pathogenic process, with recent data revealing why and offering a new strategy by which the deleterious RNA might be directly targeted. Likewise, a compelling potential therapeutic avenue for spinal muscular atrophy is illuminated by a new study in which small molecules are shown to selectively control alternative splicing of the survival of motor neuron 2 gene and ameliorate disease symptoms in mice.