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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Gepubliceerd in:Hum Mol Genet
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Oxford University Press 2019
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General Article
Online toegang: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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