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Article

The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models

4 Jun 2013

Abstract excerpt

Spinal muscular atrophy (SMA) is caused by insufficient levels of the survival motor neuron (SMN) protein due to the functional loss of the SMN1 gene and the inability of its paralog, SMN2, to fully compensate due to reduced exon 7 splicing efficiency. Since SMA patients have at least one copy of SMN2, drug discovery campaigns have sought to identify SMN2 inducers. C5-substituted quinazolines increase SMN2...

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PMID: 23736298
DOI: 10.1093/hmg/ddt258

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