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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...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Dis Model Mech |
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| Κύριοι συγγραφείς: | , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
The Company of Biologists Ltd
2017
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5560066/ https://ncbi.nlm.nih.gov/pubmed/28768735 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1242/dmm.030148 |
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