Evidence map›Paper›PMID 38408879›Full record

ReviewTrends in molecular medicine2024

HDAC inhibitors as pharmacological treatment for Duchenne muscular dystrophy: a discovery journey from bench to patients.

Chiara Mozzetta, Vittorio Sartorelli, Christian Steinkuhler, Pier Lorenzo Puri

Erratum issuedOpen access · greenAbstract readReview
In one paragraph

Review in Trends in molecular medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 37 papers.

0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed
8.9field-weighted citation impact, top 1% 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

37 citing papers in PubMed, 38 citations in OpenAlex.

  1. Article
  2. Article
  3. The histone deacetylase family in health and disease.Signal transduction and targeted therapy · 2026
    Review
  4. Article
  5. Review
  6. Article
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  8. HDAC6 orchestrates NK cell maturation and antitumor immunity.Journal for immunotherapy of cancer · 2026
    Article
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  11. Article
  12. Review
  13. Observational
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  17. Article
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

4 authors at 3 institutions in 2 countries.

Chiara MozzettaInstitute of Molecular Biology and Pathology (IBPM), National Research Council (CNR) of Italy, Rome, Italy.
Vittorio SartorelliLaboratory of Muscle Stem Cells and Gene Regulation, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), National Institutes of Health (NIH), Bethesda, MD, USA.
Christian SteinkuhlerNew Drug Incubator, Italfarmaco SpA, Milan, Italy.
Pier Lorenzo PuriDevelopment, Aging, and Regeneration Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, USA. Electronic address: lpuri@sbpdiscovery.org.
Institute of Molecular Biology and Pathology · ITNational Institutes of Health · USSanford Burnham Prebys Medical Discovery Institute · US

Funding

Pathogenic Alterations of the 3D Epigenetic Landscape in Dystrophin-Deficient Skeletal Muscles and Reversal by Dystrophin Re-ExpressionR01AR056712 · NIAMS · SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE · PI Pier Lorenzo Puri · 2009 to 2026
$7.3M
MYOD Regulation of 3D Chromatin StructureR01GM134712 · NIGMS · SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE · PI PURI, PIER LORENZO · 2019 to 2022
$1.6M
NIAMS NIH HHS R01 AR056712NIGMS NIH HHS R01 GM134712
6 · The paper itself

Abstract

Earlier evidence that targeting the balance between histone acetyltransferases (HATs) and deacetylases (HDACs), through exposure to HDAC inhibitors (HDACis), could enhance skeletal myogenesis, prompted interest in using HDACis to promote muscle regeneration. Further identification of constitutive HDAC activation in dystrophin-deficient muscles, caused by dysregulated nitric oxide (NO) signaling, provided the rationale for HDACi-based therapeutic interventions for Duchenne muscular dystrophy (DMD). In this review, we describe the molecular, preclinical, and clinical evidence supporting the efficacy of HDACis in countering disease progression by targeting pathogenic networks of gene expression in multiple muscle-resident cell types of patients with DMD. Given that givinostat is paving the way for HDACi-based interventions in DMD, next-generation HDACis with optimized therapeutic profiles and efficacy could be also explored for synergistic combinations with other therapeutic strategies.

Indexed as

Muscular Dystrophy, DuchenneAnimalsDystrophinHistone Deacetylase InhibitorsHumansMiceMice, Inbred mdxMuscle, SkeletalSignal TransductionDystrophinHistone Deacetylase Inhibitorsduchenne muscular dystrophyfibro-adipogenic degenerationHDAC inhibitorshistone acetylationmuscle regeneration

Identifiers

PMID38408879
PMCPMC11095976
OpenAlexW4392170446

What OpenQuestion holds

Textmetadata
LicenceTDM
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.