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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Detalhes bibliográficos
Main Authors: Zanetta, Chiara, Riboldi, Giulietta, Nizzardo, Monica, Simone, Chiara, Faravelli, Irene, Bresolin, Nereo, Comi, Giacomo P, Corti, Stefania
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons Ltd 2014
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3930406/
https://ncbi.nlm.nih.gov/pubmed/24400925
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jcmm.12224
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