ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease and one of the leading inherited causes of infant mortality. SMA results from insufficient levels of the survival motor neuron (SMN) protein, and studies in animal models of the disease have shown that increasing SMN prote...

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Dades bibliogràfiques
Publicat a:JCI Insight
Autors principals: Abera, Mahlet B., Xiao, Jingbo, Nofziger, Jonathan, Titus, Steve, Southall, Noel, Zheng, Wei, Moritz, Kasey E., Ferrer, Marc, Cherry, Jonathan J., Androphy, Elliot J., Wang, Amy, Xu, Xin, Austin, Christopher, Fischbeck, Kenneth H., Marugan, Juan J., Burnett, Barrington G.
Format: Artigo
Idioma:Inglês
Publicat: American Society for Clinical Investigation 2016
Matèries:
Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5111506/
https://ncbi.nlm.nih.gov/pubmed/27882347
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.88427
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