Evidence map›Paper›PMID 33349909›Full record

ReviewStem cell reviews and reports2021

Systemic cell therapy for muscular dystrophies : The ultimate transplantable muscle progenitor cell and current challenges for clinical efficacy.

C Rosanne M Ausems, Baziel G M van Engelen, Hans van Bokhoven, Derick G Wansink

Open access · hybridAbstract readReview
In one paragraph

Review in Stem cell reviews and reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
0.8field-weighted citation impact, top 30% 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

16 citing papers in PubMed, 16 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Zika virus infection disturbs development of human muscle progenitor cells.Frontiers in cellular and infection microbiology · 2025
    Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Article
  11. Review
  12. Article
  13. Current Strategies of Muscular Dystrophy Therapeutics: An Overview.Methods in molecular biology (Clifton, N.J.) · 2023
    Review
  14. Article
  15. Article
  16. 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 1 institution in 2 countries.

C Rosanne M AusemsDonders lnstitute for Brain Cognition and Behavior, Department of Human Genetics, Radboud University Medical Center, 6525, GA, Nijmegen, The Netherlands.
Baziel G M van EngelenDonders lnstitute for Brain Cognition and Behavior, Department of Neurology, Radboud University Medical Center, 6525, GA, Nijmegen, The Netherlands.
Hans van Bokhoven *Donders lnstitute for Brain Cognition and Behavior, Department of Human Genetics, Radboud University Medical Center, 6525, GA, Nijmegen, The Netherlands. hans.vanbokhoven@radboudumc.nl.
Derick G Wansink *Radboud Institute for Molecular Life Sciences, Department of Cell Biology, Radboud University Medical Center, 6525, GA, Nijmegen, The Netherlands. rick.wansink@radboudumc.nl.ORCID 0000-0002-6773-8662
Radboud University Nijmegen · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The intrinsic regenerative capacity of skeletal muscle makes it an excellent target for cell therapy. However, the potential of muscle tissue to renew is typically exhausted and insufficient in muscular dystrophies (MDs), a large group of heterogeneous genetic disorders showing progressive loss of skeletal muscle fibers. Cell therapy for MDs has to rely on suppletion with donor cells with high myogenic regenerative capacity. Here, we provide an overview on stem cell lineages employed for strategies in MDs, with a focus on adult stem cells and progenitor cells resident in skeletal muscle. In the early days, the potential of myoblasts and satellite cells was explored, but after disappointing clinical results the field moved to other muscle progenitor cells, each with its own advantages and disadvantages. Most recently, mesoangioblasts and pericytes have been pursued for muscle cell therapy, leading to a handful of preclinical studies and a clinical trial. The current status of (pre)clinical work for the most common forms of MD illustrates the existing challenges and bottlenecks. Besides the intrinsic properties of transplantable cells, we discuss issues relating to cell expansion and cell viability after transplantation, optimal dosage, and route and timing of administration. Since MDs are genetic conditions, autologous cell therapy and gene therapy will need to go hand-in-hand, bringing in additional complications. Finally, we discuss determinants for optimization of future clinical trials for muscle cell therapy. Joined research efforts bring hope that effective therapies for MDs are on the horizon to fulfil the unmet clinical need in patients.

Indexed as

Muscular DystrophiesCell- and Tissue-Based TherapyHumansMusclesMyoblastsStem CellsTreatment OutcomeCell therapyGene therapyMesoangioblastMuscle regenerationMuscle stem cellMuscular dystrophyMyogenic progenitor cellPericyteSatellite cellSkeletal muscle

Identifiers

PMID33349909
PMCPMC8166694
OpenAlexW3115202675

What OpenQuestion 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 OpenQuestion graph.