Myostatin: a Circulating Biomarker Correlating with Disease in Myotubular Myopathy Mice and Patients

Myotubular myopathy, also called X-linked centronuclear myopathy (XL-CNM), is a severe congenital disease targeted for therapeutic trials. To date, biomarkers to monitor disease progression and therapy efficacy are lacking. The Mtm1(−/y) mouse is a faithful model for XL-CNM, due to myotubularin 1 (M...

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Veröffentlicht in:Mol Ther Methods Clin Dev
Hauptverfasser: Koch, Catherine, Buono, Suzie, Menuet, Alexia, Robé, Anne, Djeddi, Sarah, Kretz, Christine, Gomez-Oca, Raquel, Depla, Marion, Monseur, Arnaud, Thielemans, Leen, Servais, Laurent, Laporte, Jocelyn, Cowling, Belinda S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2020
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7267729/
https://ncbi.nlm.nih.gov/pubmed/32514412
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.022
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