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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Detalhes bibliográficos
Publicado no:Dis Model Mech
Main Authors: Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Formato: Artigo
Idioma:Inglês
Publicado em: The Company of Biologists Ltd 2017
Assuntos:
Review
Acesso em linha: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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