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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Salvato in:
Dettagli Bibliografici
Pubblicato in:Dis Model Mech
Autori principali: Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Natura: Artigo
Lingua:Inglês
Pubblicazione: The Company of Biologists Ltd 2017
Soggetti:
Review
Accesso 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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