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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...

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Detalles Bibliográficos
Autores principales: Magri, Francesca, Vanoli, Fiammetta, Corti, Stefania
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
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