A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy Controls

BACKGROUND: Clinical trials to test safety and efficacy of drugs for patients with spinal muscular atrophy (SMA) are currently underway. Biomarkers that document treatment-induced effects are needed because disease progression in childhood forms of SMA is slow and clinical outcome measures may lack...

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Veröffentlicht in:PLoS One
Hauptverfasser: Wadman, Renske I., Stam, Marloes, Jansen, Marc D., van der Weegen, Yana, Wijngaarde, Camiel A., Harschnitz, Oliver, Sodaar, Peter, Braun, Kees P. J., Dooijes, Dennis, Lemmink, Henny H., van den Berg, Leonard H., van der Pol, W. Ludo
Format: Artigo
Sprache:Inglês
Veröffentlicht: Public Library of Science 2016
Schlagworte:
Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5125671/
https://ncbi.nlm.nih.gov/pubmed/27893852
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0167087
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