RHO and proximal spinal muscular atrophy: Our pathway analyses revealed that many molecular functions that are dysregulated in SMA mice compared with WT mice and recovered by Pip6a-PMO have previously been implicated in the pathology of SMA, including RNA metabolism and splicing, circadian regulation of gene expression, ubiquitin pathways, regulation of Rho protein signal transduction, and actin-binding pathways (51–54).