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Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)

Spinal muscular atrophy (SMA) is an autosomal recessive motor neuron disease. It is the first genetic cause of infant mortality. It is caused by mutations in the survival motor neuron 1 (SMN1) gene, leading to the reduction of SMN protein. The most striking component is the loss of alpha motor neuro...

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Detalles Bibliográficos
Autores principales:	Zanetta, Chiara, Riboldi, Giulietta, Nizzardo, Monica, Simone, Chiara, Faravelli, Irene, Bresolin, Nereo, Comi, Giacomo P, Corti, Stefania
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons Ltd 2014
Materias:	Reviews
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3930406/ https://www.ncbi.nlm.nih.gov/pubmed/24400925 http://dx.doi.org/10.1111/jcmm.12224

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