The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models

Spinal muscular atrophy (SMA) is caused by insufficient levels of the survival motor neuron (SMN) protein due to the functional loss of the SMN1 gene and the inability of its paralog, SMN2, to fully compensate due to reduced exon 7 splicing efficiency. Since SMA patients have at least one copy of SM...

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Autori principali: Gogliotti, Rocky G., Cardona, Herminio, Singh, Jasbir, Bail, Sophie, Emery, Carina, Kuntz, Nancy, Jorgensen, Michael, Durens, Madel, Xia, Bing, Barlow, Courtenay, Heier, Christopher R., Plasterer, Heather L., Jacques, Vincent, Kiledjian, Megerditch, Jarecki, Jill, Rusche, James, DiDonato, Christine J.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2013
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Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3781638/
https://ncbi.nlm.nih.gov/pubmed/23736298
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddt258
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