Lookup NU author(s): Lauren Phillips,
Professor Volker Straub
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© 2018 Future Medicine Ltd. 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.
Author(s): Catapano F, Domingos J, Perry M, Ricotti V, Phillips L, Servais L, Seferian A, Groot ID, Krom YD, Niks EH, Verschuuren JJGM, Straub V, Voit T, Morgan J, Muntoni F
Publication type: Article
Publication status: Published
Online publication date: 22/03/2018
Acceptance date: 02/04/2018
ISSN (print): 1750-1911
ISSN (electronic): 1750-192X
Publisher: Future Medicine Ltd.
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