Open access
Jul 2026
Antisense oligonucleotides treatment uncovers differences in the modulation of dysregulated intracellular pathways in Spinal Muscular Atrophy motoneurons
Observations indicate that nusinersen-like ASO treatment might be insufficient to counteract the full spectrum of intracellular alterations occurring in SMN-reduced MNs, and supplementary compounds targeting these unrecovered pathways might supply additional protective effects on degenerating MNs.
María P. Miralles, Isabel Gimenez-Fernandez, María Beltran et al.
· Cell Death and Disease · 0 citations