Evidence map›Paper›PMID 28252048›Full record

ArticleScientific reports2017

Identification of serum protein biomarkers for utrophin based DMD therapy.

Simon Guiraud, Benjamin Edwards, Sarah E Squire, Arran Babbs, Nandini Shah, Adam Berg, Huijia Chen, Kay E Davies

Open access · goldAbstract read
In one paragraph

Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
2.6field-weighted citation impact, top 10% 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

20 citing papers in PubMed, 37 citations in OpenAlex.

  1. Serum protein biomarker signature of Duchenne muscular dystrophy.European journal of translational myology · 2025
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  12. The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2020
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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

8 authors at 3 institutions in 1 country.

Simon GuiraudMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Benjamin EdwardsMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Sarah E SquireMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Arran BabbsMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Nandini ShahMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Adam BergMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Huijia ChenMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
Kay E DaviesMedical Research Council Functional Genomics Unit at the University of Oxford, Department of Physiology, Anatomy and Genetics, Oxford, OX1 3PT, United Kingdom.
University of Oxford · GBMedical Research Council · GBGenomics (United Kingdom) · GB

Funding

Medical Research Council MR/N010698/1
6 · The paper itself

Abstract

Despite promising therapeutic avenues, there is currently no effective treatment for Duchenne muscular dystrophy (DMD), a lethal monogenic disorder caused by the loss of the large cytoskeletal protein, dystrophin. A highly promising approach to therapy, applicable to all DMD patients irrespective to their genetic defect, is to modulate utrophin, a functional paralogue of dystrophin, able to compensate for the primary defects of DMD restoring sarcolemmal stability. One of the major difficulties in assessing the effectiveness of therapeutic strategies is to define appropriate outcome measures. In the present study, we utilised an aptamer based proteomics approach to profile 1,310 proteins in plasma of wild-type, mdx and Fiona (mdx overexpressing utrophin) mice. Comparison of the C57 and mdx sera revealed 83 proteins with statistically significant >2 fold changes in dystrophic serum abundance. A large majority of previously described biomarkers (ANP32B, THBS4, CAMK2A/B/D, CYCS, CAPNI) were normalised towards wild-type levels in Fiona animals. This work also identified potential mdx markers specific to increased utrophin (DUS3, TPI1) and highlights novel mdx biomarkers (GITR, MYBPC1, HSP60, SIRT2, SMAD3, CNTN1). We define a panel of putative protein mdx biomarkers to evaluate utrophin based strategies which may help to accelerate their translation to the clinic.

Indexed as

BiomarkersBlood ProteinsAnimalsDisease Models, AnimalEnzyme-Linked Immunosorbent AssayHumansMiceMice, TransgenicMuscular Dystrophy, AnimalMuscular Dystrophy, DuchenneProteomeProteomicsTranslational Research, BiomedicalUtrophinBiomarkersBlood ProteinsProteomeUtrophin

Identifiers

PMID28252048
PMCPMC5333102
OpenAlexW2593353214

What OpenQuestion holds

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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 OpenQuestion graph.