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miRNA in spinal muscular atrophy pathogenesis and therapy
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease characterized by the selective death of lower motor neurons in the brain stem and spinal cord. SMA is caused by mutations in the survival motor neuron 1 gene (SMN1), leading to the reduced expression of the full‐length...
Autores principales: | Magri, Francesca, Vanoli, Fiammetta, Corti, Stefania |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5783860/ https://www.ncbi.nlm.nih.gov/pubmed/29160009 http://dx.doi.org/10.1111/jcmm.13450 |
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