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Article

<i>SMN2</i> splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

7 Aug 2014

Abstract excerpt

Spinal muscular atrophy (SMA) is a genetic disease caused by mutation or deletion of the survival of motor neuron 1 (SMN1) gene. A paralogous gene in humans, SMN2, produces low, insufficient levels of functional SMN protein due to alternative splicing that truncates the transcript. The decreased levels of SMN protein lead to progressive neuromuscular degeneration and high rates of mortality. Through chemical...

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PMID: 25104390
DOI: 10.1126/science.1250127

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