Evidence map›Paper›PMID 31546754›Full record

ReviewInternational journal of molecular sciences2019

miRNA Profiling for Early Detection and Treatment of Duchenne Muscular Dystrophy.

Heather C Hrach, Marco Mangone

Open access · goldAbstract readReview
In one paragraph

Review in International journal of molecular sciences, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed, 1 pooled it
1.8field-weighted citation impact, top 14% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed, 1 synthesis or guideline pooled it, 37 citations in OpenAlex.

  1. Pooled it
  2. CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
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  4. Differential responses to avian pathogenicFrontiers in cellular and infection microbiology · 2024
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors at 1 institution in 1 country.

Heather C HrachMolecular and Cellular Biology Graduate Program, School of Life Sciences 427 East Tyler Mall, Tempe, AZ 85287 4501, USA. Heather.Geissel@asu.edu.
Marco MangoneVirginia G. Piper Center for Personalized Diagnostics, The Biodesign Institute at Arizona State University, 1001 S McAllister Ave, Tempe, AZ 85287, USA. mangone@asu.edu.
Arizona State University · US

Funding

Genetics and Genomics of Alternative Polyadenylation and miRNA Regulation in C. e - Renewal - 1R01GM118796 · NIGMS · ARIZONA STATE UNIVERSITY-TEMPE CAMPUS · PI MANGONE, MARCO · 2016 to 2024
$2.8M
NIGMS NIH HHS 1R01GM118796NIGMS NIH HHS R01 GM118796
6 · The paper itself

Abstract

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.

Indexed as

Cell-Free Nucleic AcidsGene Expression ProfilingMicroRNAsMuscular Dystrophy, DuchenneBiomarkersEarly DiagnosisHumansBiomarkersCell-Free Nucleic AcidsMicroRNAsbiomarkercirculating miRNAsDuchenne muscular dystrophy

Identifiers

PMID31546754
PMCPMC6769970
OpenAlexW2973972555

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.