Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches

Spinal muscular atrophy (SMA) is a primary genetic cause of infant mortality due to mutations in the Survival Motor Neuron (SMN) 1 gene. No cure is available. Antisense oligonucleotides (ASOs) aimed at increasing SMN levels from the paralogous SMN2 gene represent a possible therapeutic strategy. Her...

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Publicat a:Sci Rep
Autors principals: Nizzardo, Monica, Simone, Chiara, Dametti, Sara, Salani, Sabrina, Ulzi, Gianna, Pagliarani, Serena, Rizzo, Federica, Frattini, Emanuele, Pagani, Franco, Bresolin, Nereo, Comi, Giacomo, Corti, Stefania
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2015
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4485234/
https://ncbi.nlm.nih.gov/pubmed/26123042
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep11746
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