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Spinal muscular atrophy (SMA) is a degenerative motor neuron disease caused by homozygous mutations of the survival motor neuron 1 (SMN1) gene. Effective treatment for SMA is unavailable at present. The aim of this study was to investigate the effect of hydroxyurea (HU) in SMA cells and patients.Fifteen SMA lymphoid and three fibroblast cell lines, 2 from SMA patients and 1 control, were treated with...
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