Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a leading genetic cause of infantile death and is caused by the loss of survival motor neuron-1 (SMN1). Importantly, a nearly identical gene is present called SMN2; however, the majority of SMN2-derived transcripts are alternatively spliced and encode a truncated, dy...

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Pubblicato in:JCI Insight
Autori principali: Kaifer, Kevin A., Villalón, Eric, Osman, Erkan Y., Glascock, Jacqueline J., Arnold, Laura L., Cornelison, D.D.W., Lorson, Christian L.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society for Clinical Investigation 2017
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5333955/
https://ncbi.nlm.nih.gov/pubmed/28289706
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.89970
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