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The Survival of Motor Neurons (SMN) protein participates in RNA splicing. Decreases in SMN, typically a consequence of defects in the smn1 gene, result in the death of motor neurons and lead to the neurodegenerative disease, spinal muscular atrophy (SMA). Cuspin-1 is a small molecule upregulator of SMN that has been shown in vitro to increase levels of SMN in SMA patient fibroblasts by 50% at 18 μM. Its mechanism of action is thought to involve increased phosphorylation of ERK to initiate Ras-Raf-MEK signaling, which results in an increased rate of SMN translation.

Cuspin 1 is used in cuspin-1 characterization and regulation of SMN protein via Ras signaling in spinal muscular atropy.

[1] REKA R. LETSO. Small Molecule Screen Reveals Regulation of Survival Motor Neuron Protein Abundance by Ras Proteins[J]. ACS Chemical Biology, 2013, 8 5: 914-922. DOI: 10.1021/cb300374h