A severe mouse model of spinal muscular atrophy develops early systemic inflammation

Abstract excerpt

Spinal muscular atrophy (SMA) is a fatal genetic disease, mainly affecting children. A number of recent studies show, aside from lower motor neuron degeneration and atrophy of skeletal muscles, widespread defects present in the central nervous system (CNS) and peripheral non-neuronal cell types of SMA patients and mouse models, particularly of severe forms. However, molecular mechanisms underlying the multi-organ...

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