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Paper Details

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
Journal of Medicinal Chemistry
63
2016
RG7800, SMA, SMN protein, SMN1, SMN2, SMN2 mRNA, SMN7 protein, Spinal Muscular Atrophy, Spinal muscular atrophy, Survival, Survival Motor Neuron 2, exon 7, full-length SMN protein, full-length SMN2 mRNA, human, infant, mouse, pyridopyrimidinone, survival motor neuron 1, survival motor neuron 1 (SMN1) gene, truncated

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