Stat5 constitutive activation rescues defects in spinal muscular atrophy.

@article{Ting2007Stat5CA,
  title={Stat5 constitutive activation rescues defects in spinal muscular atrophy.},
  author={Chen-Hung Ting and Chiao-Wei Lin and Shin-Lan Wen and Hsiu-Mei Hsieh-Li and Hung Li},
  journal={Human molecular genetics},
  year={2007},
  volume={16 5},
  pages={499-514}
}
Proximal spinal muscular atrophy (SMA) is a motor neuron degeneration disorder for which there is currently no effective treatment. Here, we report three compounds (sodium vanadate, trichostatin A and aclarubicin) that effectively enhance SMN2 expression by inducing Stat5 activation in SMA-like mouse embryonic fibroblasts and human SMN2-transfected NSC34 cells. We found that Stat5 activation enhanced SMN2 promoter activity with increase in both full-length and deletion exon 7 SMN transcripts in… CONTINUE READING
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