ReviewNature reviews. Genetics2013
Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.
Review in Nature reviews. Genetics, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 127 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
127 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Genome Editing of Monogenic Neuromuscular Diseases: A Systematic Review.JAMA neurology · 2016Pooled it
- Mapping Autoantibodies in Children With Acute Rheumatic Fever.Frontiers in immunology · 2021Trial
- Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy.Journal of clinical pharmacology · 2019Trial
- Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.Annals of neurology · 2016Trial
- Engineering a compact CgCas12n platform for efficient genome and base editing in mammalian cells.Molecular systems biology · 2026Article
- Swallowing Dysfunction and Histopathological Changes in a Rat Model of Duchenne Muscular Dystrophy.Dysphagia · 2026Article
- AI-powered therapeutic aptamer drug discovery: Targeting the CT-domain of CTGF for duchenne muscular dystrophy.Acta pharmaceutica Sinica. B · 2026Article
- Current and Emerging Therapeutic Strategies for the Treatment of Duchenne Muscular Dystrophy.Genes · 2026Review
- Assessing off-target effects in CRISPR/Cas9: challenges and strategies for precision DNA editing.Archives of microbiology · 2026Review
- Improvement of Spontaneous Locomotor Activity in a Murine Model of Duchenne Muscular Dystrophy by N-Acetylglucosamine Alone and in Combination With Prednisolone.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2025Article
- Anti-fibrotic, muscle-promoting antibody-drug conjugates for the improvement and treatment of DMD.iScience · 2025Article
- Development of a local controlled release system for therapeutic proteins in the treatment of skeletal muscle injuries and diseases.Cell death & disease · 2024Article
- Decreasing microtubule detyrosination modulates Nav1.5 subcellular distribution and restores sodium current in mdx cardiomyocytes.Cardiovascular research · 2024Article
- Antisense Oligonucleotide-Mediated Downregulation of IGFBPs Enhances IGF-1 Signaling.Journal of neuromuscular diseases · 2024Article
- Type-B monoamine oxidase inhibitors in neurological diseases: clinical applications based on preclinical findings.Neural regeneration research · 2024Review
- Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients.Stem cell research · 2023Article
- Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors.Molecular therapy. Nucleic acids · 2023Article
- Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs.Stem cell reports · 2023Article
- Overexpression of synaptic vesicle protein Rab GTPase 3C promotes vesicular exocytosis and drug resistance in colorectal cancer cells.Molecular oncology · 2023Article
67 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatment except palliative therapy. There are several promising genetic approaches, including viral delivery of the missing dystrophin gene, read-through of translation stop codons, exon skipping to restore the reading frame and increased expression of the compensatory utrophin gene. The lessons learned from these approaches will be applicable to many other disorders.
Indexed as
Identifiers
23609411What OpenQuestion holds
Registered trials
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.