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Spinal muscular atrophy (SMA) is caused by the reduced expression of the survival of motor neuron (SMN) protein due to the loss of functional SMN1 gene and alternative splicing of exon 7 in the SMN2 gene. We are pursuing innovative drug discovery strategies aimed at restoring the production of the SMN protein by modulating SMN2 alternative splicing. Panels of cell based assays and animal models have...
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