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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Dis Model Mech
Prif Awduron: Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: The Company of Biologists Ltd 2017
Pynciau:
Review
Mynediad Ar-lein: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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