Immune Cell-Specific SMN Expression and Sustained Immune Alterations in Treated Spinal Muscular Atrophy.
Mutations in the survival of motor neuron 1 (SMN1) can reduce functional SMN protein levels, which causes Spinal Muscular Atrophy (SMA), a disease affecting the nervous system and peripheral tissues, including the immune system. Yet, SMN expression across immune cell subsets and the impact of SMN-modulating therapies o...