Therapeutic strategies for spinal muscular atrophy: SMN and beyond
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor neurons and muscle atrophy, generally presenting in childhood. SMA is caused by low levels of the survival motor neuron protein (SMN) due to inactivating mutations in the encoding gene SMN1. A second...
| Auteurs principaux: | , , , , , , , |
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| Format: | Journal article |
| Publié: |
Company of Biologists
2017
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