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miRNA Profiling for Early Detection and Treatment of Duchenne Muscular Dystrophy

Molecular and Cellular Biology Graduate Program, School of Life Sciences 427 East Tyler Mall, Tempe, AZ 85287 4501, USA
Virginia G. Piper Center for Personalized Diagnostics, The Biodesign Institute at Arizona State University, 1001 S McAllister Ave, Tempe, AZ 85287, USA
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2019, 20(18), 4638;
Received: 23 August 2019 / Revised: 10 September 2019 / Accepted: 17 September 2019 / Published: 19 September 2019
(This article belongs to the Special Issue New Regulators and Modulators of MicroRNA)
Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder caused by out of frame mutations in the dystrophin gene. The hallmark symptoms of the condition include progressive degeneration of skeletal muscle, cardiomyopathy, and respiratory dysfunction. The most recent advances in therapeutic strategies for the treatment of DMD involve exon skipping or administration of minidystrophin, but these strategies are not yet universally available, nor have they proven to be a definitive cure for all DMD patients. Early diagnosis and tracking of symptom progression of DMD usually relies on creatine kinase tests, evaluation of patient performance in various ambulatory assessments, and detection of dystrophin from muscle biopsies, which are invasive and painful for the patient. While the current research focuses primarily on restoring functional dystrophin, accurate and minimally invasive methods to detect and track both symptom progression and the success of early DMD treatments are not yet available. In recent years, several groups have identified miRNA signature changes in DMD tissue samples, and a number of promising studies consistently detected changes in circulating miRNAs in blood samples of DMD patients. These results could potentially lead to non-invasive detection methods, new molecular approaches to treating DMD symptoms, and new methods to monitor of the efficacy of the therapy. In this review, we focus on the role of circulating miRNAs in DMD and highlight their potential both as a biomarker in the early detection of disease and as a therapeutic target in the prevention and treatment of DMD symptoms. View Full-Text
Keywords: circulating miRNAs; Duchenne muscular dystrophy; biomarker circulating miRNAs; Duchenne muscular dystrophy; biomarker
MDPI and ACS Style

Hrach, H.C.; Mangone, M. miRNA Profiling for Early Detection and Treatment of Duchenne Muscular Dystrophy. Int. J. Mol. Sci. 2019, 20, 4638.

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