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Small molecule-induced regulation of the SMN2 splicing mechanism. (a) The splicing mechanism of SMN1 (healthy protein) and SMN2 (disease protein) in which Exon 7 is included due to a SNP. (b) Small molecules shown to induce Exon 7 stabilization in SMN2 gene. Abbreviations: SNP, single nucleotide polymorphism; SMN, survival motor neuron; ESE, exonic splicing enhancer; hnRNP G, heterogeneous nuclear ribonucleoprotein G; snRNP, small nuclear ribonucleoprotein; ss, splice site.
