Evidence mapPaperPMID 36056021Full record

ArticleNPJ Regenerative medicine2022

Transplantation of PSC-derived myogenic progenitors counteracts disease phenotypes in FSHD mice.

Karim Azzag, Darko Bosnakovski, Sudheer Tungtur, Peter Salama, Michael Kyba, Rita C R Perlingeiro

Open access · goldAbstract read
In one paragraph

Article in NPJ Regenerative medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.8field-weighted citation impact, top 31% 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

10 citing papers in PubMed, 9 citations in OpenAlex.

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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

6 authors at 1 institution in 1 country.

Karim AzzagLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
Darko BosnakovskiLillehei Heart Institute, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Sudheer TungturLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
Peter SalamaLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
Michael KybaLillehei Heart Institute, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Rita C R PerlingeiroLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA. perli032@umn.edu.ORCID http://orcid.org/0000-0001-9412-1118
University of Minnesota · US

Funding

Probing the activity of DUX4 in FSHDR01AR055685 · UNIVERSITY OF MINNESOTA · 2025 to 2025
$518k
Skeletal Muscle Regeneration from Pluripotent Stem CellsR01AR078571 · UNIVERSITY OF MINNESOTA · 2025 to 2025
$409k
NIAMS NIH HHS R01 AR055685NIAMS NIH HHS R01 AR071439NIAMS NIH HHS R01 AR078571
6 · The paper itself

Abstract

Facioscapulohumeral muscular dystrophy (FSHD) is a genetically dominant progressive myopathy caused by improper silencing of the DUX4 gene, leading to fibrosis, muscle atrophy, and fatty replacement. Approaches focused on muscle regeneration through the delivery of stem cells represent an attractive therapeutic option for muscular dystrophies. To investigate the potential for cell transplantation in FSHD, we have used the doxycycline-regulated iDUX4pA-HSA mouse model in which low-level DUX4 can be induced in skeletal muscle. We find that mouse pluripotent stem cell (PSC)-derived myogenic progenitors engraft in muscle actively undergoing DUX4-mediated degeneration. Donor-derived muscle tissue displayed reduced fibrosis and importantly, engrafted muscles showed improved contractile specific force compared to non-transplanted controls. These data demonstrate the feasibility of replacement of diseased muscle with PSC-derived myogenic progenitors in a mouse model for FSHD, and highlight the potential for the clinical benefit of such a cell therapy approach.

Identifiers

PMID36056021
PMCPMC9440030
OpenAlexW4294225706

What Socratic 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 Socratic graph.