Evidence map›Paper›PMID 32695843›Full record

ArticleMolecular therapy. Methods & clinical development2020

Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy.

Virginie Mariot, Caroline Le Guiner, Inès Barthélémy, Marie Montus, Stéphane Blot, Silvia Torelli, Jennifer Morgan, Francesco Muntoni, Thomas Voit, Julie Dumonceaux

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
1.3field-weighted citation impact, top 21% 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

14 citing papers in PubMed, 26 citations in OpenAlex.

  1. Article
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  8. Replenishing NADSkeletal muscle · 2023
    Article
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  13. Therapeutic Strategies Targeting DUX4 in FSHD.Journal of clinical medicine · 2020
    Review
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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

10 authors at 6 institutions in 2 countries.

Virginie MariotNIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital NHS Trust, London WC1N 1EH, UK.
Caroline Le GuinerINSERM UMR 1089, Université de Nantes, CHU de Nantes, IRS2 Nantes Biotech, 22, bd Bénoni Goullin, 44200 Nantes, France.
Inès BarthélémyU955-IMRB, Team 10, Biology of the Neuromuscular System, INSERM, UPEC, EFS, Ecole Nationale Vétérinaire d'Alfort, 94700 Maisons-Alfort, France.
Marie MontusGénéthon, 1 bis rue de l'Internationale, 91000 Evry, France.
Stéphane BlotU955-IMRB, Team 10, Biology of the Neuromuscular System, INSERM, UPEC, EFS, Ecole Nationale Vétérinaire d'Alfort, 94700 Maisons-Alfort, France.
Silvia TorelliNIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital NHS Trust, London WC1N 1EH, UK.
Jennifer MorganNIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital NHS Trust, London WC1N 1EH, UK.
Francesco MuntoniNIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital NHS Trust, London WC1N 1EH, UK.
Thomas VoitNIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital NHS Trust, London WC1N 1EH, UK.
Julie DumonceauxNIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health, and Great Ormond Street Hospital NHS Trust, London WC1N 1EH, UK.
University College London · GBGreat Ormond Street Hospital · GBCentre Hospitalier Universitaire de Nantes · FRÉcole Nationale Vétérinaire d'Alfort · FRGenethon (France) · FRInserm · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recently, several promising treatments have emerged for neuromuscular disorders, highlighting the need for robust biomarkers for monitoring therapeutic efficacy and maintenance of the therapeutic effect. Several studies have proposed circulating and tissue biomarkers, but none of them has been validated to monitor acute and long-term drug response. We previously described how the myostatin (MSTN) level is naturally downregulated in several neuromuscular diseases, including Duchenne muscular dystrophy (DMD). Here, we show that the dystrophin-deficient Golden Retriever muscular dystrophy (GRMD) dog model also presents an intrinsic loss of Mstn production in muscle. The abnormally low levels of Mstn observed in the GRMD dog puppies at 2 months were partially rescued at both mRNA and protein level after adeno-associated virus (AAV)-microdystrophin treatment in a dose-dependent manner. These results show that circulating Mstn is a robust and reliable quantitative biomarker, capable of measuring a therapeutic response to pharmaco-gene therapy in real time in the neuromuscular system, as well as a quantitative means for non-invasive follow-up of a therapeutic effect. Moreover, a 2-year follow-up also suggests that Mstn could be a longitudinal monitoring tool to follow maintenance or decrease of the therapeutic effect.

Indexed as

biomarkerclinical trialsDuchenne muscular dystrophy (DMD)GDF8myostatinneuromuscular disorderstherapy

Identifiers

PMID32695843
PMCPMC7363622
OpenAlexW3036354469

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.