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A Negatively Acting Bifunctional RNA Increases <i>Survival Motor Neuron</i> Both <i>In Vitro</i> and <i>In Vivo</i>

1 Nov 2008

Abstract excerpt

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder and is the leading genetic cause of infant mortality. SMA is caused by the loss of survival motor neuron-1 (SMN1). In humans, a nearly identical copy gene is present called SMN2, but this gene cannot compensate for the loss of SMN1 because of a single silent nucleotide difference in SMN2 exon 7. This single-nucleotide difference...

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PMID: 19848583
DOI: 10.1089/hum.2008.067

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A Negatively Acting Bifunctional RNA Increases <i>Survival Motor Neuron</i> Both <i>In Vitro</i> and <i>In Vivo</i>PMID 19848583Read ↗

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