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Establishing a baseline and drug-induced SMN expression profile in SMA disease-relevant human tissues in expedited autopsies

drug-induced512

Spinal muscular atrophy (SMA) is a recessive neuromuscular disease that is caused by loss-of-function mutations in the survival motor neuron 1 (SMN1) gene and is one of the most common inheritable causes of infant death. In a recent study, researchers investigated baseline and drug-induced SMN levels in disease-relevant human tissue, find out what they learned.

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Establishing a baseline and drug-induced SMN expression profile in SMA disease-relevant human tissues in expedited autopsies

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