MiR34 Contributes to Spinal Muscular Atrophy and AAV9-mediated Delivery of MiR34a Ameliorates the Motor Deficits in SMA Mice

MiR34 exhibits consistent downregulation during SMA progression in both human and rodent contexts. Mir34 family-knockout mice display axon swelling and reduced neuromuscular junction (NMJ) end-plates, recapitulating SMA pathology. MiR34 family may target synapse formation and several core pathways that are disrupted in SMA at early postnatal stages. We performed RNA seq on thoracic and lumbar spinal cord and compare gene expression profiles between P14 wild type (WT) and Mir34/449 triple knockout (TKO) mice. Comparative gene expression profiling analysis of RNA-seq data for WT and TKO spinal cord samples.