Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.

<h4>Background</h4>Collagen VI related myopathies encompass a range of phenotypes with involvement of skeletal muscle, skin and other connective tissues. They represent a severe and relatively common form of congenital disease for which there is no treatment. Collagen VI in skeletal musc...

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Main Authors: Sonia Paco, Teresa Casserras, Maria Angels Rodríguez, Cristina Jou, Montserrat Puigdelloses, Carlos I Ortez, Jordi Diaz-Manera, Eduardo Gallardo, Jaume Colomer, Andrés Nascimento, Susana G Kalko, Cecilia Jimenez-Mallebrera
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2015-01-01
Series:PLoS ONE
Online Access:https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0145107&type=printable
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author Sonia Paco
Teresa Casserras
Maria Angels Rodríguez
Cristina Jou
Montserrat Puigdelloses
Carlos I Ortez
Jordi Diaz-Manera
Eduardo Gallardo
Jaume Colomer
Andrés Nascimento
Susana G Kalko
Cecilia Jimenez-Mallebrera
author_facet Sonia Paco
Teresa Casserras
Maria Angels Rodríguez
Cristina Jou
Montserrat Puigdelloses
Carlos I Ortez
Jordi Diaz-Manera
Eduardo Gallardo
Jaume Colomer
Andrés Nascimento
Susana G Kalko
Cecilia Jimenez-Mallebrera
author_sort Sonia Paco
collection DOAJ
description <h4>Background</h4>Collagen VI related myopathies encompass a range of phenotypes with involvement of skeletal muscle, skin and other connective tissues. They represent a severe and relatively common form of congenital disease for which there is no treatment. Collagen VI in skeletal muscle and skin is produced by fibroblasts.<h4>Aims & methods</h4>In order to gain insight into the consequences of collagen VI mutations and identify key disease pathways we performed global gene expression analysis of dermal fibroblasts from patients with Ullrich Congenital Muscular Dystrophy with and without vitamin C treatment. The expression data were integrated using a range of systems biology tools. Results were validated by real-time PCR, western blotting and functional assays.<h4>Findings</h4>We found significant changes in the expression levels of almost 600 genes between collagen VI deficient and control fibroblasts. Highly regulated genes included extracellular matrix components and surface receptors, including integrins, indicating a shift in the interaction between the cell and its environment. This was accompanied by a significant increase in fibroblasts adhesion to laminin. The observed changes in gene expression profiling may be under the control of two miRNAs, miR-30c and miR-181a, which we found elevated in tissue and serum from patients and which could represent novel biomarkers for muscular dystrophy. Finally, the response to vitamin C of collagen VI mutated fibroblasts significantly differed from healthy fibroblasts. Vitamin C treatment was able to revert the expression of some key genes to levels found in control cells raising the possibility of a beneficial effect of vitamin C as a modulator of some of the pathological aspects of collagen VI related diseases.
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spelling doaj-art-1e761410602c489094dc77afc19dbdbc2025-08-20T02:15:41ZengPublic Library of Science (PLoS)PLoS ONE1932-62032015-01-011012e014510710.1371/journal.pone.0145107Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.Sonia PacoTeresa CasserrasMaria Angels RodríguezCristina JouMontserrat PuigdellosesCarlos I OrtezJordi Diaz-ManeraEduardo GallardoJaume ColomerAndrés NascimentoSusana G KalkoCecilia Jimenez-Mallebrera<h4>Background</h4>Collagen VI related myopathies encompass a range of phenotypes with involvement of skeletal muscle, skin and other connective tissues. They represent a severe and relatively common form of congenital disease for which there is no treatment. Collagen VI in skeletal muscle and skin is produced by fibroblasts.<h4>Aims & methods</h4>In order to gain insight into the consequences of collagen VI mutations and identify key disease pathways we performed global gene expression analysis of dermal fibroblasts from patients with Ullrich Congenital Muscular Dystrophy with and without vitamin C treatment. The expression data were integrated using a range of systems biology tools. Results were validated by real-time PCR, western blotting and functional assays.<h4>Findings</h4>We found significant changes in the expression levels of almost 600 genes between collagen VI deficient and control fibroblasts. Highly regulated genes included extracellular matrix components and surface receptors, including integrins, indicating a shift in the interaction between the cell and its environment. This was accompanied by a significant increase in fibroblasts adhesion to laminin. The observed changes in gene expression profiling may be under the control of two miRNAs, miR-30c and miR-181a, which we found elevated in tissue and serum from patients and which could represent novel biomarkers for muscular dystrophy. Finally, the response to vitamin C of collagen VI mutated fibroblasts significantly differed from healthy fibroblasts. Vitamin C treatment was able to revert the expression of some key genes to levels found in control cells raising the possibility of a beneficial effect of vitamin C as a modulator of some of the pathological aspects of collagen VI related diseases.https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0145107&type=printable
spellingShingle Sonia Paco
Teresa Casserras
Maria Angels Rodríguez
Cristina Jou
Montserrat Puigdelloses
Carlos I Ortez
Jordi Diaz-Manera
Eduardo Gallardo
Jaume Colomer
Andrés Nascimento
Susana G Kalko
Cecilia Jimenez-Mallebrera
Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.
PLoS ONE
title Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.
title_full Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.
title_fullStr Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.
title_full_unstemmed Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.
title_short Transcriptome Analysis of Ullrich Congenital Muscular Dystrophy Fibroblasts Reveals a Disease Extracellular Matrix Signature and Key Molecular Regulators.
title_sort transcriptome analysis of ullrich congenital muscular dystrophy fibroblasts reveals a disease extracellular matrix signature and key molecular regulators
url https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0145107&type=printable
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