Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy

X-linked myotubular myopathy (XLMTM) is a severe muscle disease with no effective treatment. Here, the authors show that tamoxifen, a drug used to treat breast cancer, rescues the pathology in a mouse model of the disease, at least in part by normalizing expression of the disease modifier proteins D...

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Autores principales: Elinam Gayi, Laurence A. Neff, Xènia Massana Muñoz, Hesham M. Ismail, Marta Sierra, Thomas Mercier, Laurent A. Décosterd, Jocelyn Laporte, Belinda S. Cowling, Olivier M. Dorchies, Leonardo Scapozza
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Lenguaje:EN
Publicado: Nature Portfolio 2018
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Acceso en línea:https://doaj.org/article/55e31f0f62ce4d928b4e2b13a82b1d60
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spelling oai:doaj.org-article:55e31f0f62ce4d928b4e2b13a82b1d602021-12-02T14:41:28ZTamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy10.1038/s41467-018-07058-42041-1723https://doaj.org/article/55e31f0f62ce4d928b4e2b13a82b1d602018-11-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-07058-4https://doaj.org/toc/2041-1723X-linked myotubular myopathy (XLMTM) is a severe muscle disease with no effective treatment. Here, the authors show that tamoxifen, a drug used to treat breast cancer, rescues the pathology in a mouse model of the disease, at least in part by normalizing expression of the disease modifier proteins DNM2 and BIN1Elinam GayiLaurence A. NeffXènia Massana MuñozHesham M. IsmailMarta SierraThomas MercierLaurent A. DécosterdJocelyn LaporteBelinda S. CowlingOlivier M. DorchiesLeonardo ScapozzaNature PortfolioarticleScienceQENNature Communications, Vol 9, Iss 1, Pp 1-14 (2018)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Elinam Gayi
Laurence A. Neff
Xènia Massana Muñoz
Hesham M. Ismail
Marta Sierra
Thomas Mercier
Laurent A. Décosterd
Jocelyn Laporte
Belinda S. Cowling
Olivier M. Dorchies
Leonardo Scapozza
Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
description X-linked myotubular myopathy (XLMTM) is a severe muscle disease with no effective treatment. Here, the authors show that tamoxifen, a drug used to treat breast cancer, rescues the pathology in a mouse model of the disease, at least in part by normalizing expression of the disease modifier proteins DNM2 and BIN1
format article
author Elinam Gayi
Laurence A. Neff
Xènia Massana Muñoz
Hesham M. Ismail
Marta Sierra
Thomas Mercier
Laurent A. Décosterd
Jocelyn Laporte
Belinda S. Cowling
Olivier M. Dorchies
Leonardo Scapozza
author_facet Elinam Gayi
Laurence A. Neff
Xènia Massana Muñoz
Hesham M. Ismail
Marta Sierra
Thomas Mercier
Laurent A. Décosterd
Jocelyn Laporte
Belinda S. Cowling
Olivier M. Dorchies
Leonardo Scapozza
author_sort Elinam Gayi
title Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
title_short Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
title_full Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
title_fullStr Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
title_full_unstemmed Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy
title_sort tamoxifen prolongs survival and alleviates symptoms in mice with fatal x-linked myotubular myopathy
publisher Nature Portfolio
publishDate 2018
url https://doaj.org/article/55e31f0f62ce4d928b4e2b13a82b1d60
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