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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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2016
Assuntos:
Research Article
Acesso em linha: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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