ReviewExperimental physiology2015
Advances in genetic therapeutic strategies for Duchenne muscular dystrophy.
Review in Experimental physiology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 papers.
What it found
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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
38 citing papers in PubMed, 80 citations in OpenAlex.
- Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study.Annals of clinical and translational neurology · 2020Trial
- Multiple modes of AFM reveal distinct mechanical properties for dystrophin and utrophin not manifest by small fragments.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
- Dystrophin Restorative and Compensatory Gene Addition Therapies for Duchenne Muscular Dystrophy: Could CRISPRa Provide a Realistic Alternative?Muscles (Basel, Switzerland) · 2025Review
- Development of a local controlled release system for therapeutic proteins in the treatment of skeletal muscle injuries and diseases.Cell death & disease · 2024Article
- Two operational modes of atomic force microscopy reveal similar mechanical properties for homologous regions of dystrophin and utrophin.bioRxiv : the preprint server for biology · 2024Article
- Histone deacetylase inhibition with givinostat: a multi-targeted mode of action with the potential to halt the pathological cascade of Duchenne muscular dystrophy.Frontiers in cell and developmental biology · 2024Review
- Pharmacotherapeutic Approaches to Treatment of Muscular Dystrophies.Biomolecules · 2023Review
- Real-world evidence of eteplirsen treatment effects in patients with Duchenne muscular dystrophy in the USA.Journal of comparative effectiveness research · 2023Article
- Step-by-Step Double-Trouble OBAIRH and DMD Diagnosis in a One-Year-Old Boy.International journal of molecular sciences · 2023Article
- Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up.Stem cell reviews and reports · 2023Article
- Clinical application of next generation sequencing-based haplotype linkage analysis in the preimplantation genetic testing for germline mosaicisms.Orphanet journal of rare diseases · 2023Article
- Preliminary Results of a Genetic Study of Children with Duchenne Myodystrophy in Aktobe, Kazakhstan.Archives of Razi Institute · 2023Article
- Continuitiy of care with ataluren in Duchenne Muscular Dystrophy patients with nonsense mutations after loss of ambulation. Personal experience.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2023Article
- Inhibition of PKCθ Improves Dystrophic Heart Phenotype and Function in a Novel Model of DMD Cardiomyopathy.International journal of molecular sciences · 2022Article
- Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy.Frontiers in medicine · 2022Review
- Targeting IRES-dependent translation as a novel approach for treating Duchenne muscular dystrophy.RNA biology · 2021Review
- Sonoelastography of the trunk and lower extremity muscles in a case of Duchenne muscular dystrophy.Journal of ultrasound · 2021Review
- Dystrophin gene editing by CRISPR/Cas9 system in human skeletal muscle cell line (HSkMC).Iranian journal of basic medical sciences · 2021Article
- Catalpol counteracts the pathology in a mouse model of Duchenne muscular dystrophy by inhibiting the TGF-β1/TAK1 signaling pathway.Acta pharmacologica Sinica · 2021Article
- Clinical Evaluation of Duchenne Muscular Dystrophy Severity Using Ultrasound Small-Window Entropy Imaging.Entropy (Basel, Switzerland) · 2020Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
NEW
findingsWhat is the topic of this review? This review highlights recent progress in genetically based therapies targeting the primary defect of Duchenne muscular dystrophy. What advances does it highlight? Over the last two decades, considerable progress has been made in understanding the mechanisms underlying Duchenne muscular dystrophy, leading to the development of genetic therapies. These include manipulation of the expression of the gene or related genes, the splicing of the gene and its translation, and replacement of the gene using viral approaches. Duchenne muscular dystrophy is a lethal X-linked disorder caused by mutations in the dystrophin gene. In the absence of the dystrophin protein, the link between the cytoskeleton and extracellular matrix is destroyed, and this severely compromises the strength, flexibility and stability of muscle fibres. The devastating consequence is progressive muscle wasting and premature death in Duchenne muscular dystrophy patients. There is currently no cure, and despite exhaustive palliative care, patients are restricted to a wheelchair by the age of 12 years and usually succumb to cardiac or respiratory complications in their late 20s. This review provides an update on the current genetically based therapies and clinical trials that target or compensate for the primary defect of this disease. These include dystrophin gene-replacement strategies, genetic modification techniques to restore dystrophin expression, and modulation of the dystrophin homologue, utrophin, as a surrogate to re-establish muscle function.
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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.