DM1: an oligonucleotide administered in the brain of model mice

• IONIS 486178, an antisense oligonucleotide optimised to target muscle cells (through coupling to an antibody) injected systemically, improves muscle damage in mice with DM1.
• Intracerebroventricular injection reduces behavioural impairment in treated mice.
• These results open up the possibility of intrathecal treatment in DM1.

Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1. Ait Benichou S. et al.  Gene Ther. 2022 Jan