ASO Molecular Therapies May Be an ALS Treatment Strategy

In a collaboration between Wave Life Sciences and UMass Medical School, scientists aimed to target RNA molecules that contain the G4C2 expansion using tiny stereopure ASO molecules, which bind to RNA.With this approach, investigators intended to prevent the accumulation of the abnormal molecules while avoiding changes in C9orf72 protein levels.They then confirmed the activity of these ASO sequences in patient-derived fibroblasts and in mouse neurons under a microscope.Researchers optimized the ASO chemistry, which resulted in potent, stereopure ASOs, as confirmed in the activity assays.“These results suggest that preferential targeting of repeat-containing transcripts using stereopure ASOs may be a viable therapeutic approach for the treatment of ALS and [frontotemporal dementia],” the researchers wrote.
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