Evidence map›Paper›PMID 31639376›Full record

ReviewExperimental neurology2020

Stem cell-based therapies for Duchenne muscular dystrophy.

Congshan Sun, Carlo Serra, Gabsang Lee, Kathryn R Wagner

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Experimental neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
60citing papers in PubMed, 1 pooled it
5.7field-weighted citation impact, top 3% 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

60 citing papers in PubMed, 1 synthesis or guideline pooled it, 104 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Observational
  4. Article
  5. Review
  6. Review
  7. Article
  8. Article
  9. Article
  10. Stem/progenitor cell-based therapy for Duchenne muscular dystrophy.Frontiers in cell and developmental biology · 2025
    Review
  11. Developing Advanced Chimeric Cell Therapy for Duchenne Muscular Dystrophy.International journal of molecular sciences · 2024
    Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Review
  17. Article
  18. Article
  19. Article
  20. Article
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

4 authors at 3 institutions in 1 country.

Congshan SunDepartments of Neurology and Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA; Center for Genetic Muscle Disorders, Hugo W. Moser Research Institute at Kennedy Krieger Institute, Baltimore, MD 21205, USA. Electronic address: csun23@jhmi.edu.
Carlo SerraDepartments of Neurology and Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA; Center for Genetic Muscle Disorders, Hugo W. Moser Research Institute at Kennedy Krieger Institute, Baltimore, MD 21205, USA.
Gabsang LeeInstitute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
Kathryn R WagnerDepartments of Neurology and Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, 21205, USA; Center for Genetic Muscle Disorders, Hugo W. Moser Research Institute at Kennedy Krieger Institute, Baltimore, MD 21205, USA.
Johns Hopkins Medicine · USJohns Hopkins University · USKennedy Krieger Institute · US

Funding

Interrogating functional and molecular properties of PAX7+ putative skeletal muscle stem/progenitor cells derived from human iPSCs of healthy donors and Duchenne muscular dystrophy patientsR01AR070751 · NIAMS · JOHNS HOPKINS UNIVERSITY · PI LEE, GABSANG · 2017 to 2021
$1.8M
Cell extrinsic factors' roles on direct conversion to human induced neural crestR01NS093213 · NINDS · JOHNS HOPKINS UNIVERSITY · PI LEE, GABSANG · 2015 to 2019
$1.8M
NIAMS NIH HHS R01 AR070751NINDS NIH HHS R01 NS093213
6 · The paper itself

Abstract

Muscular dystrophies are a group of genetic muscle disorders that cause progressive muscle weakness and degeneration. Within this group, Duchenne muscular dystrophy (DMD) is the most common and one of the most severe. DMD is an X chromosome linked disease that occurs to 1 in 3500 to 1 in 5000 boys. The cause of DMD is a mutation in the dystrophin gene, whose encoded protein provides both structural support and cell signaling capabilities. So far, there are very limited therapeutic options available and there is no cure for this disease. In this review, we discuss the existing cell therapy research, especially stem cell-based, which utilize myoblasts, satellite cells, bone marrow cells, mesoangioblasts and CD133+ cells. Finally, we focus on human pluripotent stem cells (hPSCs) which hold great potential in treating DMD. hPSCs can be used for autologous transplantation after being specified to a myogenic lineage. Over the last few years, there has been a rapid development of isolation, as well as differentiation, techniques in order to achieve effective transplantation results of myogenic cells specified from hPSCs. In this review, we summarize the current methods of hPSCs myogenic commitment/differentiation, and describe the current status of hPSC-derived myogenic cell transplantation.

Indexed as

Cell DifferentiationHumansMuscular Dystrophy, DuchenneMyoblastsPluripotent Stem CellsStem Cell TransplantationDuchenne muscular dystrophy (DMD)hiPSCMyogenic lineage specificationStem cell therapy

Identifiers

PMID31639376
PMCPMC6899334
OpenAlexW2980523496

What OpenQuestion holds

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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.