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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Autors principals: Zanetta, Chiara, Riboldi, Giulietta, Nizzardo, Monica, Simone, Chiara, Faravelli, Irene, Bresolin, Nereo, Comi, Giacomo P, Corti, Stefania
Format: Artigo
Idioma:Inglês
Publicat: John Wiley & Sons Ltd 2014
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Reviews
Accés en línia: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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