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Article

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models

29 Apr 2014

Abstract excerpt

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by the loss of Survival Motor Neuron-1 (SMN1). In all SMA patients, a nearly identical copy gene called SMN2 is present, which produces low levels of functional protein owing to an alternative splicing event. To prevent exon-skipping, we have targeted an intronic repressor, Element1 (E1), located upstream of SMN2 exon 7 using Morpholino-based...

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PMID: 24781211
DOI: 10.1093/hmg/ddu198

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