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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Dis Model Mech
Päätekijät: Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: The Company of Biologists Ltd 2017
Aiheet:
Review
Linkit: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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