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...
| Главные авторы: | , , , , , , , |
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| Формат: | Journal article |
| Опубликовано: |
Company of Biologists
2017
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Therapeutic strategies for spinal muscular atrophy: SMN and beyond
Опубликовано 2017
Journal article