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Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies

The development of medical approaches requires preclinical and clinical trials for assessment of therapeutic efficacy. Such evaluation entails the use of biomarkers, which provide information on the response to the therapeutic intervention. One newly-proposed class of biomarkers is the microRNA (miR...

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Autores principales: Israeli, David, Poupiot, Jérôme, Amor, Fatima, Charton, Karine, Lostal, William, Jeanson-Leh, Laurence, Richard, Isabelle
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4914855/
https://www.ncbi.nlm.nih.gov/pubmed/27323895
http://dx.doi.org/10.1038/srep28097
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author Israeli, David
Poupiot, Jérôme
Amor, Fatima
Charton, Karine
Lostal, William
Jeanson-Leh, Laurence
Richard, Isabelle
author_facet Israeli, David
Poupiot, Jérôme
Amor, Fatima
Charton, Karine
Lostal, William
Jeanson-Leh, Laurence
Richard, Isabelle
author_sort Israeli, David
collection PubMed
description The development of medical approaches requires preclinical and clinical trials for assessment of therapeutic efficacy. Such evaluation entails the use of biomarkers, which provide information on the response to the therapeutic intervention. One newly-proposed class of biomarkers is the microRNA (miRNA) molecules. In muscular dystrophies (MD), the dysregulation of miRNAs was initially observed in muscle biopsy and later extended to plasma samples, suggesting that they may be of interest as biomarkers. First, we demonstrated that dystromiRs dysregulation occurs in MD with either preserved or disrupted expression of the dystrophin-associated glycoprotein complex, supporting the utilization of dystromiRs as generic biomarkers in MD. Then, we aimed at evaluation of the capacity of miRNAs as monitoring biomarkers for experimental therapeutic approach in MD. To this end, we took advantage of our previously characterized gene therapy approach in a mouse model for α-sarcoglycanopathy. We identified a dose-response correlation between the expression of miRNAs on both muscle tissue and blood serum and the therapeutic benefit as evaluated by a set of new and classically-used evaluation methods. This study supports the utility of profiling circulating miRNAs for the evaluation of therapeutic outcome in medical approaches for MD.
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spelling pubmed-49148552016-06-27 Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies Israeli, David Poupiot, Jérôme Amor, Fatima Charton, Karine Lostal, William Jeanson-Leh, Laurence Richard, Isabelle Sci Rep Article The development of medical approaches requires preclinical and clinical trials for assessment of therapeutic efficacy. Such evaluation entails the use of biomarkers, which provide information on the response to the therapeutic intervention. One newly-proposed class of biomarkers is the microRNA (miRNA) molecules. In muscular dystrophies (MD), the dysregulation of miRNAs was initially observed in muscle biopsy and later extended to plasma samples, suggesting that they may be of interest as biomarkers. First, we demonstrated that dystromiRs dysregulation occurs in MD with either preserved or disrupted expression of the dystrophin-associated glycoprotein complex, supporting the utilization of dystromiRs as generic biomarkers in MD. Then, we aimed at evaluation of the capacity of miRNAs as monitoring biomarkers for experimental therapeutic approach in MD. To this end, we took advantage of our previously characterized gene therapy approach in a mouse model for α-sarcoglycanopathy. We identified a dose-response correlation between the expression of miRNAs on both muscle tissue and blood serum and the therapeutic benefit as evaluated by a set of new and classically-used evaluation methods. This study supports the utility of profiling circulating miRNAs for the evaluation of therapeutic outcome in medical approaches for MD. Nature Publishing Group 2016-06-21 /pmc/articles/PMC4914855/ /pubmed/27323895 http://dx.doi.org/10.1038/srep28097 Text en Copyright © 2016, Macmillan Publishers Limited http://creativecommons.org/licenses/by/4.0/ This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
spellingShingle Article
Israeli, David
Poupiot, Jérôme
Amor, Fatima
Charton, Karine
Lostal, William
Jeanson-Leh, Laurence
Richard, Isabelle
Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
title Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
title_full Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
title_fullStr Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
title_full_unstemmed Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
title_short Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
title_sort circulating mirnas are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4914855/
https://www.ncbi.nlm.nih.gov/pubmed/27323895
http://dx.doi.org/10.1038/srep28097
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