Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

Drugs targeting myostatin reverse muscle wasting in animal models, but have limited efficacy in patients. The authors show that the myostatin pathway is downregulated in patients, possibly explaining the poor outcome of anti-myostatin approaches, and that it can be reactivated by correcting disease-...

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Autores principales:	Virginie Mariot, Romain Joubert, Christophe Hourdé, Léonard Féasson, Michael Hanna, Francesco Muntoni, Thierry Maisonobe, Laurent Servais, Caroline Bogni, Rozen Le Panse, Olivier Benvensite, Tanya Stojkovic, Pedro M. Machado, Thomas Voit, Ana Buj-Bello, Julie Dumonceaux
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2017
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/2403fa3958844630ae86e529ecfe4061
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spelling	oai:doaj.org-article:2403fa3958844630ae86e529ecfe40612021-12-02T14:40:26ZDownregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches10.1038/s41467-017-01486-42041-1723https://doaj.org/article/2403fa3958844630ae86e529ecfe40612017-11-01T00:00:00Zhttps://doi.org/10.1038/s41467-017-01486-4https://doaj.org/toc/2041-1723Drugs targeting myostatin reverse muscle wasting in animal models, but have limited efficacy in patients. The authors show that the myostatin pathway is downregulated in patients, possibly explaining the poor outcome of anti-myostatin approaches, and that it can be reactivated by correcting disease-causing mutations in mice.Virginie MariotRomain JoubertChristophe HourdéLéonard FéassonMichael HannaFrancesco MuntoniThierry MaisonobeLaurent ServaisCaroline BogniRozen Le PanseOlivier BenvensiteTanya StojkovicPedro M. MachadoThomas VoitAna Buj-BelloJulie DumonceauxNature PortfolioarticleScienceQENNature Communications, Vol 8, Iss 1, Pp 1-8 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Virginie Mariot Romain Joubert Christophe Hourdé Léonard Féasson Michael Hanna Francesco Muntoni Thierry Maisonobe Laurent Servais Caroline Bogni Rozen Le Panse Olivier Benvensite Tanya Stojkovic Pedro M. Machado Thomas Voit Ana Buj-Bello Julie Dumonceaux Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
description	Drugs targeting myostatin reverse muscle wasting in animal models, but have limited efficacy in patients. The authors show that the myostatin pathway is downregulated in patients, possibly explaining the poor outcome of anti-myostatin approaches, and that it can be reactivated by correcting disease-causing mutations in mice.
format	article
author	Virginie Mariot Romain Joubert Christophe Hourdé Léonard Féasson Michael Hanna Francesco Muntoni Thierry Maisonobe Laurent Servais Caroline Bogni Rozen Le Panse Olivier Benvensite Tanya Stojkovic Pedro M. Machado Thomas Voit Ana Buj-Bello Julie Dumonceaux
author_facet	Virginie Mariot Romain Joubert Christophe Hourdé Léonard Féasson Michael Hanna Francesco Muntoni Thierry Maisonobe Laurent Servais Caroline Bogni Rozen Le Panse Olivier Benvensite Tanya Stojkovic Pedro M. Machado Thomas Voit Ana Buj-Bello Julie Dumonceaux
author_sort	Virginie Mariot
title	Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
title_short	Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
title_full	Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
title_fullStr	Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
title_full_unstemmed	Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
title_sort	downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
publisher	Nature Portfolio
publishDate	2017
url	https://doaj.org/article/2403fa3958844630ae86e529ecfe4061
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Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

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