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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Bibliografiska uppgifter
Huvudupphovsmän: Zanetta, Chiara, Riboldi, Giulietta, Nizzardo, Monica, Simone, Chiara, Faravelli, Irene, Bresolin, Nereo, Comi, Giacomo P, Corti, Stefania
Materialtyp: Artigo
Språk:Inglês
Publicerad: John Wiley & Sons Ltd 2014
Ämnen:
Reviews
Länkar: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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