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 languageEnglish
JournalEpigenomics
Early online date22 Mar 2018
DOIs
Publication statusEarly online - 22 Mar 2018

Keywords

  • biomarker
  • deflazacort
  • Duchenne muscular dystrophy
  • exosome
  • mirRNA
  • prednisolone

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