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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Detalles Bibliográficos
Autores principales: Bowerman, Melissa, Becker, Catherina G., Yáñez-Muñoz, Rafael J., Ning, Ke, Wood, Matthew J. A., Gillingwater, Thomas H., Talbot, Kevin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2017
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5560066/
https://www.ncbi.nlm.nih.gov/pubmed/28768735
http://dx.doi.org/10.1242/dmm.030148

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5560066/
https://www.ncbi.nlm.nih.gov/pubmed/28768735
http://dx.doi.org/10.1242/dmm.030148

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