Nature Communications (Nov 2017)
Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
- 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
Affiliations
- Virginie Mariot
- NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust
- Romain Joubert
- Genethon, INSERM U951, Univ Evry, Université Paris-Saclay
- Christophe Hourdé
- Inter-University Laboratory of Human Movement Biology (LIBM)–EA7424 Université Savoie Mont Blanc, Campus Scientifique Technolac
- Léonard Féasson
- Laboratoire Interuniversitaire de Biologie de la Motricité–EA 7424, Univ Lyon, UJM-Saint-Etienne, Unité de Myologie, Centre Référent Maladies Neuromusculaires Rares Rhône-Alpes, CHU Saint-Etienne
- Michael Hanna
- Department of Molecular Neuroscience, MRC Centre for Neuromuscular Diseases, Institute of Neurology, University College London
- Francesco Muntoni
- NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust
- Thierry Maisonobe
- Groupe Hospitalier Pitié-Salpêtrière Département de Neurophysiologie Clinique
- Laurent Servais
- I-Motion–Research Center for Pediatric Neuromuscular Diseases, Armand Trousseau Hospital
- Caroline Bogni
- Genethon, INSERM U951, Univ Evry, Université Paris-Saclay
- Rozen Le Panse
- Sorbonne Universités, UPMC Univ Paris 06, Centre de Recherche en Myologie, INSERM UMRS974, Institut de Myologie
- Olivier Benvensite
- Sorbonne Universités, UPMC Univ Paris 06, Centre de Recherche en Myologie, INSERM UMRS974, Institut de Myologie
- Tanya Stojkovic
- Centre de référence de Pathologie Neuromusculaire Paris-Est, Institut de Myologie, GHU Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris
- Pedro M. Machado
- Department of Molecular Neuroscience, MRC Centre for Neuromuscular Diseases, Institute of Neurology, University College London
- Thomas Voit
- NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust
- Ana Buj-Bello
- Genethon, INSERM U951, Univ Evry, Université Paris-Saclay
- Julie Dumonceaux
- NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust
- DOI
- https://doi.org/10.1038/s41467-017-01486-4
- Journal volume & issue
-
Vol. 8,
no. 1
pp. 1 – 8
Abstract
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.
