Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients

Francesco Catapano; Joana Domingos; Mark Perry; Valeria Ricotti; Lauren Phillips; Laurent Servais; Andreea Seferian; Imelda de Groot; Yvonne D. Krom; Erik H. Niks; Jan Jgm Verschuuren; Volker Straub; Thomas Voit; Jennifer Morgan; Francesco Muntoni

doi:10.2217/epi-2018-0022

Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients

Francesco Catapano, Joana Domingos, Mark Perry, Valeria Ricotti, Lauren Phillips, Laurent Servais, Andreea Seferian, Imelda de Groot, Yvonne D. Krom, Erik H. Niks, Jan Jgm Verschuuren, Volker Straub, Thomas Voit, Jennifer Morgan, Francesco Muntoni

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Abstract

Aim: To study the signature of 87 urinary miRNAs in Duchenne muscular dystrophy (DMD) patients, select the most dysregulated and determine statistically significant differences in their expression between controls, ambulant (A) and nonambulant (NA) DMD patients, and patients on different corticosteroid regimens.

Patients/materials & methods: Urine was collected from control (n = 20), A (n = 31) and NA (n = 23) DMD patients. miRNA expression was measured by reverse transcription-quantitative PCR.

Results: miR-29c-3p was significantly downregulated in A DMD patients while miR-23b-3p and miR-21-5p were significantly downregulated in NA DMD patients compared with age-matched controls.

Conclusion: miR-29c-3p, miR-23b-3p and miR-21-5p are promising novel noninvasive biomarkers for DMD, and miR-29c-3p levels are differentially affected by different steroid regimens, supporting the antifibrotic effect of steroid therapy.

Original language	English
Journal	Epigenomics
Early online date	22 Mar 2018
DOIs	https://doi.org/10.2217/epi-2018-0022
Publication status	Early online - 22 Mar 2018

Keywords

biomarker
deflazacort
Duchenne muscular dystrophy
exosome
mirRNA
prednisolone

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10.2217/epi-2018-0022

Downregulation_of_microRNA_29
Francesco Catapano, Joana Domingos, Mark Perry, Valeria Ricotti, Lauren Phillips, Laurent Servais, Andreea Seferian, Imelda de Groot, Yvonne D Krom, Erik H Niks, Jan JGM Verschuuren, Volker Straub, Thomas Voit, Jennifer Morgan, and Francesco Muntoni, 'Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients,' Epigenomics.
Accepted author manuscript (Post-print), 816 KB

https://www.futuremedicine.com/doi/10.2217/epi-2018-0022

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Catapano, F., Domingos, J., Perry, M., Ricotti, V., Phillips, L., Servais, L., Seferian, A., Groot, I. D., Krom, Y. D., Niks, E. H., Verschuuren, J. J., Straub, V., Voit, T., Morgan, J., & Muntoni, F. (2018). Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients. Epigenomics. Advance online publication. https://doi.org/10.2217/epi-2018-0022

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title = "Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients",

abstract = "Aim: To study the signature of 87 urinary miRNAs in Duchenne muscular dystrophy (DMD) patients, select the most dysregulated and determine statistically significant differences in their expression between controls, ambulant (A) and nonambulant (NA) DMD patients, and patients on different corticosteroid regimens.Patients/materials & methods: Urine was collected from control (n = 20), A (n = 31) and NA (n = 23) DMD patients. miRNA expression was measured by reverse transcription-quantitative PCR. Results: miR-29c-3p was significantly downregulated in A DMD patients while miR-23b-3p and miR-21-5p were significantly downregulated in NA DMD patients compared with age-matched controls. Conclusion: miR-29c-3p, miR-23b-3p and miR-21-5p are promising novel noninvasive biomarkers for DMD, and miR-29c-3p levels are differentially affected by different steroid regimens, supporting the antifibrotic effect of steroid therapy.",

keywords = "biomarker, deflazacort, Duchenne muscular dystrophy, exosome, mirRNA, prednisolone",

author = "Francesco Catapano and Joana Domingos and Mark Perry and Valeria Ricotti and Lauren Phillips and Laurent Servais and Andreea Seferian and Groot, {Imelda de} and Krom, {Yvonne D.} and Niks, {Erik H.} and Verschuuren, {Jan Jgm} and Volker Straub and Thomas Voit and Jennifer Morgan and Francesco Muntoni",

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doi = "10.2217/epi-2018-0022",

language = "English",

journal = "Epigenomics",

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T1 - Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients

AU - Catapano, Francesco

AU - Domingos, Joana

AU - Perry, Mark

AU - Ricotti, Valeria

AU - Phillips, Lauren

AU - Servais, Laurent

AU - Seferian, Andreea

AU - Groot, Imelda de

AU - Krom, Yvonne D.

AU - Niks, Erik H.

AU - Verschuuren, Jan Jgm

AU - Straub, Volker

AU - Voit, Thomas

AU - Morgan, Jennifer

AU - Muntoni, Francesco

PY - 2018/3/22

Y1 - 2018/3/22

N2 - Aim: To study the signature of 87 urinary miRNAs in Duchenne muscular dystrophy (DMD) patients, select the most dysregulated and determine statistically significant differences in their expression between controls, ambulant (A) and nonambulant (NA) DMD patients, and patients on different corticosteroid regimens.Patients/materials & methods: Urine was collected from control (n = 20), A (n = 31) and NA (n = 23) DMD patients. miRNA expression was measured by reverse transcription-quantitative PCR. Results: miR-29c-3p was significantly downregulated in A DMD patients while miR-23b-3p and miR-21-5p were significantly downregulated in NA DMD patients compared with age-matched controls. Conclusion: miR-29c-3p, miR-23b-3p and miR-21-5p are promising novel noninvasive biomarkers for DMD, and miR-29c-3p levels are differentially affected by different steroid regimens, supporting the antifibrotic effect of steroid therapy.

AB - Aim: To study the signature of 87 urinary miRNAs in Duchenne muscular dystrophy (DMD) patients, select the most dysregulated and determine statistically significant differences in their expression between controls, ambulant (A) and nonambulant (NA) DMD patients, and patients on different corticosteroid regimens.Patients/materials & methods: Urine was collected from control (n = 20), A (n = 31) and NA (n = 23) DMD patients. miRNA expression was measured by reverse transcription-quantitative PCR. Results: miR-29c-3p was significantly downregulated in A DMD patients while miR-23b-3p and miR-21-5p were significantly downregulated in NA DMD patients compared with age-matched controls. Conclusion: miR-29c-3p, miR-23b-3p and miR-21-5p are promising novel noninvasive biomarkers for DMD, and miR-29c-3p levels are differentially affected by different steroid regimens, supporting the antifibrotic effect of steroid therapy.

KW - biomarker

KW - deflazacort

KW - Duchenne muscular dystrophy

KW - exosome

KW - mirRNA

KW - prednisolone

U2 - 10.2217/epi-2018-0022

DO - 10.2217/epi-2018-0022

M3 - Article

SN - 1750-1911

JO - Epigenomics

JF - Epigenomics

ER -

Downregulation of miRNA-29, -23 and -21 in urine of Duchenne muscular dystrophy patients

Abstract

Keywords

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