Open access
Jul 2026
Antisense oligonucleotides treatment uncovers differences in the modulation of dysregulated intracellular pathways in Spinal Muscular Atrophy motoneurons.
Spinal Muscular Atrophy (SMA) is a neuromuscular genetic disorder resulting from the mutation or deletion of the Survival Motor Neuron 1 (SMN1) gene and the reduction of the Survival Motor Neuron (SMN) protein. As a result, SMN level in SMA depends on the almost identical copy gene SMN2, which produces a small amount o...
María P. Miralles, Isabel Gimenez-Fernandez, María Beltran et al.
· Cell Death and Disease · 0 citations