Loss of motor neurons by targeted knockdown of Smn in muscle stem cells

Survival of Motor Neuron 1 (SMN1) gene encodes SMN protein and its deficit leads to spinal Muscular Atrophy (SMA).

Skeletal muscle tissue and the Muscle Stem Cells (MuSC) are affected by SMN. The researchers investigated the role of muscle stem cells in SMA.

They show that SMA type II patient muscles display a reduced number of quiescent MuSC and in SMA mice, SMN deficit compromises MuSC reservoir establishment.

The authors also demonstrate that MuSC depletion disrupts neuromuscular junctions and leads to motor neuron loss in the spinal cord.

They conclude that muscle stem cells represent potential therapeutic targets.

https://academic.oup.com/brain/advance-article/doi/10.1093/brain/awag045/8465584

https://sciencemission.com/nockdown-of-Smn-in-muscle-stem-cells