Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets

@inproceedings{Tsai2012TherapyDF,
  title={Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets},
  author={Luther Tsai},
  booktitle={Neural plasticity},
  year={2012}
}
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder, leading to progressive muscle weakness, atrophy, and sometimes premature death. SMA is caused by mutation or deletion of the survival motor neuron-1 (SMN1) gene. An effective treatment does not presently exist. Since the severity of the SMA phenotype is inversely correlated with expression levels of SMN, the SMN-encoded protein, SMN is the most important therapeutic target for development of an effective… CONTINUE READING
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