Developing Motor Neurons in SMA Show Altered Protein Production

Motor neurons derived from people with spinal muscular atrophy (SMA) showed changes in protein production at each stage of their development, from stem cells to fully mature nerve cells, a study discovered.

Findings in this reported first-such study can be used to identify protein alterations that occur before the onset of symptoms, its scientists noted. They may also be useful as new biomarkers of disease processes or patient response to treatment, or as targets for therapy candidates.

The study, “Spinal Muscular Atrophy Patient iPSC-Derived Motor Neurons Display Altered Proteomes at Early Stages of Differentiation,” was published in the journal ACS Omega.

SMA is caused by low levels of the survival motor neuron (SMN) protein due to a faulty SMN1 gene. The SMN protein plays an essential role in processing messenger RNA (mRNA), which carries instructions from genes to make proteins.

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