Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy
© 2023 The Authors. Published by Elsevier B.V. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)
| Autores: | , , , , , , , , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Tipo de recurso: | artículo |
| Estado: | Versión publicada |
| Fecha de publicación: | 2023 |
| País: | España |
| Institución: | Consejo Superior de Investigaciones Científicas (CSIC) |
| Repositorio: | DIGITAL.CSIC. Repositorio Institucional del CSIC |
| OAI Identifier: | oai:digital.csic.es:10261/351975 |
| Acceso en línea: | http://hdl.handle.net/10261/351975 |
| Access Level: | acceso abierto |
| Palabra clave: | Myostatin Follistatin Muscular dystrophy Dysferlinopathy Limb girdle muscular dystrophy R2 Miyoshi myopathy |
| id |
ES_fd7ddfa43f9976fc185fc306acb9f82a |
|---|---|
| oai_identifier_str |
oai:digital.csic.es:10261/351975 |
| network_acronym_str |
ES |
| network_name_str |
España |
| repository_id_str |
|
| spelling |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathyMoore, UrsulaFernández-Simón, EstherSchiava, MarianelaCox, DanGordish-Dressman, HeatherJames, Meredith K.Mayhew, AnnaWilson, IanGuglieri, MichelaRufibach, LauraBlamire, AndrewCarlier, Pierre G.Mori-Yoshimura, MadokaDay, John W.Jones, Kristi J.Bharucha-Goebel, Diana X.Salort-Campana, EmmanuellePestronk, AlanWalter, Maggie C.Paradas, CarmenStojkovic, TanyaBravver, ElenaPegoraro, ElenaMendell, Jerry R.Bushby, KateDíaz-Manera, JordiStraub, VolkerThe Jain COS ConsortiumMyostatinFollistatinMuscular dystrophyDysferlinopathyLimb girdle muscular dystrophy R2Miyoshi myopathy© 2023 The Authors. Published by Elsevier B.V. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)Myostatin is a myokine which acts upon skeletal muscle to inhibit growth and regeneration. Myostatin is endogenously antagonised by follistatin. This study assessed serum myostatin and follistatin concentrations as monitoring or prognostic biomarkers in dysferlinopathy, an autosomal recessively inherited muscular dystrophy. Myostatin was quantified twice with a three-year interval in 76 patients with dysferlinopathy and 38 controls. Follistatin was quantified in 62 of these patients at the same timepoints, and in 31 controls. Correlations with motor function, muscle fat fraction and contractile cross-sectional area were performed. A regression model was used to account for confounding variables. Baseline myostatin, but not follistatin, correlated with baseline function and MRI measures. However, in individual patients, three-year change in myostatin did not correlate with functional or MRI changes. Linear modelling demonstrated that function, serum creatine kinase and C-reactive protein, but not age, were independently related to myostatin concentration. Baseline myostatin concentration predicted loss of ambulation but not rate of change of functional or MRI measures, even when relative inhibition with follistatin was considered. With adjustment for extra-muscular causes of variation, myostatin could form a surrogate measure of functional ability or muscle mass, however myostatin inhibition does not form a promising treatment target in dysferlinopathy.Biomaterial was provided by the Newcastle MRC Centre Biobank for Rare & Neuromuscular Diseases, which is part of The John Walton Muscular Dystrophy Research Centre. This facility is supported by the NIHR Newcastle Biomedical Research Centre.Peer reviewedElsevierJohn Walton Centre Muscular Dystrophy Research CentreNational Institute for Health and Care Research (US)Newcastle Biomedical Research CentreConsejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]202420242023info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10261/351975reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)InglésThe underlying dataset has been published as supplementary material of the article in the publisher platform at https://doi.org/10.1016/j.nmd.2023.01.001https://doi.org/10.1016/j.nmd.2023.01.001Síinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/3519752026-05-22T06:33:51Z |
| dc.title.none.fl_str_mv |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| title |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| spellingShingle |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy Moore, Ursula Myostatin Follistatin Muscular dystrophy Dysferlinopathy Limb girdle muscular dystrophy R2 Miyoshi myopathy |
| title_short |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| title_full |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| title_fullStr |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| title_full_unstemmed |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| title_sort |
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy |
| dc.creator.none.fl_str_mv |
Moore, Ursula Fernández-Simón, Esther Schiava, Marianela Cox, Dan Gordish-Dressman, Heather James, Meredith K. Mayhew, Anna Wilson, Ian Guglieri, Michela Rufibach, Laura Blamire, Andrew Carlier, Pierre G. Mori-Yoshimura, Madoka Day, John W. Jones, Kristi J. Bharucha-Goebel, Diana X. Salort-Campana, Emmanuelle Pestronk, Alan Walter, Maggie C. Paradas, Carmen Stojkovic, Tanya Bravver, Elena Pegoraro, Elena Mendell, Jerry R. Bushby, Kate Díaz-Manera, Jordi Straub, Volker The Jain COS Consortium |
| author |
Moore, Ursula |
| author_facet |
Moore, Ursula Fernández-Simón, Esther Schiava, Marianela Cox, Dan Gordish-Dressman, Heather James, Meredith K. Mayhew, Anna Wilson, Ian Guglieri, Michela Rufibach, Laura Blamire, Andrew Carlier, Pierre G. Mori-Yoshimura, Madoka Day, John W. Jones, Kristi J. Bharucha-Goebel, Diana X. Salort-Campana, Emmanuelle Pestronk, Alan Walter, Maggie C. Paradas, Carmen Stojkovic, Tanya Bravver, Elena Pegoraro, Elena Mendell, Jerry R. Bushby, Kate Díaz-Manera, Jordi Straub, Volker The Jain COS Consortium |
| author_role |
author |
| author2 |
Fernández-Simón, Esther Schiava, Marianela Cox, Dan Gordish-Dressman, Heather James, Meredith K. Mayhew, Anna Wilson, Ian Guglieri, Michela Rufibach, Laura Blamire, Andrew Carlier, Pierre G. Mori-Yoshimura, Madoka Day, John W. Jones, Kristi J. Bharucha-Goebel, Diana X. Salort-Campana, Emmanuelle Pestronk, Alan Walter, Maggie C. Paradas, Carmen Stojkovic, Tanya Bravver, Elena Pegoraro, Elena Mendell, Jerry R. Bushby, Kate Díaz-Manera, Jordi Straub, Volker The Jain COS Consortium |
| author2_role |
author author author author author author author author author author author author author author author author author author author author author author author author author author author |
| dc.contributor.none.fl_str_mv |
John Walton Centre Muscular Dystrophy Research Centre National Institute for Health and Care Research (US) Newcastle Biomedical Research Centre Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72] |
| dc.subject.none.fl_str_mv |
Myostatin Follistatin Muscular dystrophy Dysferlinopathy Limb girdle muscular dystrophy R2 Miyoshi myopathy |
| topic |
Myostatin Follistatin Muscular dystrophy Dysferlinopathy Limb girdle muscular dystrophy R2 Miyoshi myopathy |
| description |
© 2023 The Authors. Published by Elsevier B.V. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/) |
| publishDate |
2023 |
| dc.date.none.fl_str_mv |
2023 2024 2024 |
| dc.type.none.fl_str_mv |
info:eu-repo/semantics/article http://purl.org/coar/resource_type/c_6501 Publisher's version info:eu-repo/semantics/publishedVersion |
| format |
article |
| status_str |
publishedVersion |
| dc.identifier.none.fl_str_mv |
http://hdl.handle.net/10261/351975 |
| url |
http://hdl.handle.net/10261/351975 |
| dc.language.none.fl_str_mv |
Inglés |
| language_invalid_str_mv |
Inglés |
| dc.relation.none.fl_str_mv |
The underlying dataset has been published as supplementary material of the article in the publisher platform at https://doi.org/10.1016/j.nmd.2023.01.001 https://doi.org/10.1016/j.nmd.2023.01.001 Sí |
| dc.rights.none.fl_str_mv |
info:eu-repo/semantics/openAccess |
| eu_rights_str_mv |
openAccess |
| dc.format.none.fl_str_mv |
application/pdf |
| dc.publisher.none.fl_str_mv |
Elsevier |
| publisher.none.fl_str_mv |
Elsevier |
| dc.source.none.fl_str_mv |
reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC instname:Consejo Superior de Investigaciones Científicas (CSIC) |
| instname_str |
Consejo Superior de Investigaciones Científicas (CSIC) |
| reponame_str |
DIGITAL.CSIC. Repositorio Institucional del CSIC |
| collection |
DIGITAL.CSIC. Repositorio Institucional del CSIC |
| repository.name.fl_str_mv |
|
| repository.mail.fl_str_mv |
|
| _version_ |
1869425544822521856 |
| score |
15.811543 |