Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice

X-linked myotubular myopathy is caused by mutations in the gene coding for myotubularin 1, and is characterized by overexpression of dynamin 2. Here the authors develop antisense oligonucleotides to dynamin 2, and show that systemic injection leads to improved pathology in mice.

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Autores principales: Hichem Tasfaout, Suzie Buono, Shuling Guo, Christine Kretz, Nadia Messaddeq, Sheri Booten, Sarah Greenlee, Brett P. Monia, Belinda S. Cowling, Jocelyn Laporte
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2017
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Acceso en línea:https://doaj.org/article/ea5ef61a87bf47839d28720d6a64f759
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spelling oai:doaj.org-article:ea5ef61a87bf47839d28720d6a64f7592021-12-02T14:41:59ZAntisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice10.1038/ncomms156612041-1723https://doaj.org/article/ea5ef61a87bf47839d28720d6a64f7592017-06-01T00:00:00Zhttps://doi.org/10.1038/ncomms15661https://doaj.org/toc/2041-1723X-linked myotubular myopathy is caused by mutations in the gene coding for myotubularin 1, and is characterized by overexpression of dynamin 2. Here the authors develop antisense oligonucleotides to dynamin 2, and show that systemic injection leads to improved pathology in mice.Hichem TasfaoutSuzie BuonoShuling GuoChristine KretzNadia MessaddeqSheri BootenSarah GreenleeBrett P. MoniaBelinda S. CowlingJocelyn LaporteNature PortfolioarticleScienceQENNature Communications, Vol 8, Iss 1, Pp 1-13 (2017)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Hichem Tasfaout
Suzie Buono
Shuling Guo
Christine Kretz
Nadia Messaddeq
Sheri Booten
Sarah Greenlee
Brett P. Monia
Belinda S. Cowling
Jocelyn Laporte
Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
description X-linked myotubular myopathy is caused by mutations in the gene coding for myotubularin 1, and is characterized by overexpression of dynamin 2. Here the authors develop antisense oligonucleotides to dynamin 2, and show that systemic injection leads to improved pathology in mice.
format article
author Hichem Tasfaout
Suzie Buono
Shuling Guo
Christine Kretz
Nadia Messaddeq
Sheri Booten
Sarah Greenlee
Brett P. Monia
Belinda S. Cowling
Jocelyn Laporte
author_facet Hichem Tasfaout
Suzie Buono
Shuling Guo
Christine Kretz
Nadia Messaddeq
Sheri Booten
Sarah Greenlee
Brett P. Monia
Belinda S. Cowling
Jocelyn Laporte
author_sort Hichem Tasfaout
title Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
title_short Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
title_full Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
title_fullStr Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
title_full_unstemmed Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
title_sort antisense oligonucleotide-mediated dnm2 knockdown prevents and reverts myotubular myopathy in mice
publisher Nature Portfolio
publishDate 2017
url https://doaj.org/article/ea5ef61a87bf47839d28720d6a64f759
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