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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...

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Bibliografski detalji
Izdano u:Dis Model Mech
Glavni autori: Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Format: Artigo
Jezik:Inglês
Izdano: The Company of Biologists Ltd 2017
Teme:
Online pristup: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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