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Article

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

13 Nov 2008

Abstract excerpt

In spinal muscular atrophy (SMA), the leading genetic cause of early childhood death, the survival motor neuron 1 gene (SMN1) is deleted or inactivated. The nearly identical SMN2 gene has a silent mutation that impairs the utilization of exon 7 and the production of functional protein. It has been hypothesized that therapies boosting SMN2 exon 7 inclusion might prevent or cure SMA. Exon 7 inclusion can be...

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PMID: 19010792
DOI: 10.1093/hmg/ddn382

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