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Article

Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

6 Oct 2019

Abstract excerpt

BACKGROUNDSpinal muscular atrophy (SMA) is caused by deficient expression of survival motor neuron (SMN) protein. New SMN-enhancing therapeutics are associated with variable clinical benefits. Limited knowledge of baseline and drug-induced SMN levels in disease-relevant tissues hinders efforts to optimize these treatments.METHODSSMN mRNA and protein levels were quantified in human tissues isolated during...

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PMID: 31589162
DOI: 10.1172/jci124120

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