Spinal muscular atrophy (
SMA
) is an autosomal recessive neurodegenerative disease characterized by the selective death of lower motor neurons in the brain stem and spinal cord.
SMA
is caused by mutations in the survival motor neuron 1 gene (
SMN
1
), leading to the reduced expression of the full‐length
SMN
protein. micro
RNA
s (mi
RNA
s) are small
RNA
s that regulate post‐transcriptional gene expression. Recent findings have suggested an important role for mi
RNA
s in the pathogenesis of motor neuron diseases, including
SMA
. Motor neuron‐specific mi
RNA
dysregulation in
SMA
might be implicated in their selective vulnerability. In this study, we discuss recent findings regarding the consequences of
SMN
defects on mi
RNA
s and their target
mRNA
s in motor neurons. Taken together, these data suggest that cell‐specific changes in mi
RNA
s are not only involved in the
SMA
motor neuron phenotype but can also be used as biomarkers and therapeutic targets.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.