Systems biology investigation of cAMP modulation to increase SMN levels for the treatment of spinal muscular atrophy.
Spinal muscular atrophy (SMA), a leading genetic cause of infant death worldwide, is an autosomal recessive disorder caused by the loss of SMN1 (survival motor neuron 1), which encodes the protein SMN. The loss of SMN1 causes a deficiency in SMN protein levels leading to motor neuron cell death in t...
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| Principais autores: | , , , |
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| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
Public Library of Science (PLoS)
2014-01-01
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| 叢編: | PLoS ONE |
| 在線閱讀: | http://europepmc.org/articles/PMC4267815?pdf=render |
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