AAV9-mediated delivery of miR-23a reduces disease severity in Smn(2B/−)SMA model mice

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletions or mutations in survival motor neuron 1 (SMN1). The molecular mechanisms underlying motor neuron degeneration in SMA remain elusive, as global cellular dysfunction obscures the identification and characterization of disease...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Kaifer, Kevin A, Villalón, Eric, O'Brien, Benjamin S, Sison, Samantha L, Smith, Caley E, Simon, Madeline E, Marquez, Jose, O'Day, Siri, Hopkins, Abigail E, Neff, Rachel, Rindt, Hansjörg, Ebert, Allison D, Lorson, Christian L
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
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General Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6859438/
https://ncbi.nlm.nih.gov/pubmed/31211843
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz142
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