Evidence map›Paper›PMID 32946430›Full record

ReviewThe Journal of clinical investigation2020

Stem cell therapy for muscular dystrophies.

Stefano Biressi, Antonio Filareto, Thomas A Rando

Open access · bronzeAbstract readReview
In one paragraph

Review in The Journal of clinical investigation, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 58 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
58citing papers in PubMed, 1 pooled it
4.2field-weighted citation impact, top 5% 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

58 citing papers in PubMed, 1 synthesis or guideline pooled it, 80 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Review
  5. Article
  6. Targeting C1q signaling in fibro-adipogenic progenitors prevents regenerative fibrosis of aged muscle.Proceedings of the National Academy of Sciences of the United States of America · 2026
    Article
  7. Review
  8. Review
  9. Pluripotent stem cell-based therapy toward the clinic for muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  10. Review
  11. Review
  12. Article
  13. Article
  14. Stem/progenitor cell-based therapy for Duchenne muscular dystrophy.Frontiers in cell and developmental biology · 2025
    Review
  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

3 authors at 3 institutions in 3 countries.

Stefano BiressiDepartment of Cellular, Computational and Integrative Biology (CIBIO) and.
Antonio FilaretoDepartment of Research Beyond Borders, Regenerative Medicine, Boehringer Ingelheim Pharmaceuticals Inc., Ridgefield, Conneticut, USA.
Thomas A RandoDepartment of Neurology and Neurological Sciences and.
Boehringer Ingelheim (France) · FRUniversity of Trento · ITVA Palo Alto Health Care System · US

Funding

Wnt signaling in muscle stem cell agingP01AG036695 · NIA · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Sean Curtis Bendall · 2011 to 2026
$29.3M
Molecular Regulation of Stem Cell QuiescenceR01AR073248 · NIAMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI RANDO, THOMAS A. · 2018 to 2022
$2.6M
Regulation of Muscle Fibrosis in Response to Injury and AgingI01BX002324 · VA · VETERANS ADMIN PALO ALTO HEALTH CARE SYS · PI RANDO, THOMAS A. · 2014 to 2021
–
Muscle Stem Cell Therapy for Volumetric Muscle LossI01RX001222 · VA · VETERANS ADMIN PALO ALTO HEALTH CARE SYS · PI HUANG, NGAN F. · 2014 to 2023
–
BLRD VA I01 BX002324NIAMS NIH HHS R01 AR073248NIA NIH HHS P01 AG036695RRD VA I01 RX001222
6 · The paper itself

Abstract

Muscular dystrophies are a heterogeneous group of genetic diseases, characterized by progressive degeneration of skeletal and cardiac muscle. Despite the intense investigation of different therapeutic options, a definitive treatment has not been developed for this debilitating class of pathologies. Cell-based therapies in muscular dystrophies have been pursued experimentally for the last three decades. Several cell types with different characteristics and tissues of origin, including myogenic stem and progenitor cells, stromal cells, and pluripotent stem cells, have been investigated over the years and have recently entered in the clinical arena with mixed results. In this Review, we do a roundup of the past attempts and describe the updated status of cell-based therapies aimed at counteracting the skeletal and cardiac myopathy present in dystrophic patients. We present current challenges, summarize recent progress, and make recommendations for future research and clinical trials.

Indexed as

Cell DifferentiationMuscular DystrophiesMyoblastsPluripotent Stem CellsStem Cell TransplantationHumansMuscle, SkeletalMyocardiumRegeneration

Identifiers

PMID32946430
PMCPMC7598050
OpenAlexW3087098099

What OpenQuestion holds

Textmetadata
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.