ReviewHuman genetics2016
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
Review in Human genetics, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 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.
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Who cites it
25 citing papers in PubMed, 63 citations in OpenAlex.
- CTLA4-Ig reduces proliferation and inflammatory gene expression in muscle fibroblasts, corresponding to less fibrosis and inflammation inAmerican journal of physiology. Cell physiology · 2026Article
- Respiratory characterization of a humanized Duchenne muscular dystrophy mouse model.Respiratory physiology & neurobiology · 2024Article
- Pharmacological Treatments and Therapeutic Targets in Muscle Dystrophies Generated by Alterations in Dystrophin-Associated Proteins.Medicina (Kaunas, Lithuania) · 2024Review
- Case Report: Identification of Maternal Low-Level Mosaicism in the Dystrophin Gene by Droplet Digital Polymerase Chain Reaction.Frontiers in genetics · 2021Article
- Heterogenetic parabiosis between healthy and dystrophic mice improve the histopathology in muscular dystrophy.Scientific reports · 2020Article
- Comparative proteomic analyses of Duchenne muscular dystrophy and Becker muscular dystrophy muscles: changes contributing to preserve muscle function in Becker muscular dystrophy patients.Journal of cachexia, sarcopenia and muscle · 2020Article
- Coenzyme Q10 supplementation acts as antioxidant on dystrophic muscle cells.Cell stress & chaperones · 2019Article
- Cytoplasmic nucleic acid-based XNAs directly enhance live cardiac cell function by a CaJournal of molecular and cellular cardiology · 2019Article
- Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.Nature medicine · 2019Article
- Human induced pluripotent stem cell models for the study and treatment of Duchenne and Becker muscular dystrophies.Therapeutic advances in neurological disorders · 2019Review
- Monitoring disease activity noninvasively in theProceedings of the National Academy of Sciences of the United States of America · 2018Article
- Diltiazem improves contractile properties of skeletal muscle in dysferlin-deficient BLAJ mice, but does not reduce contraction-induced muscle damage.Physiological reports · 2018Article
- Genome engineering: a new approach to gene therapy for neuromuscular disorders.Nature reviews. Neurology · 2017Review
- Structural insights on biologically relevant cationic membranes by ESR spectroscopy.Biophysical reviews · 2017Review
- Duchenne and Becker Muscular Dystrophies: A Review of Animal Models, Clinical End Points, and Biomarker Quantification.Toxicologic pathology · 2017Review
- Pediatric Cardiomyopathies.Circulation research · 2017Review
- Modulation of aberrant splicing in human RNA diseases by chemical compounds.Human genetics · 2017Review
- CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion.Genome biology · 2017Article
- FDA-Approved Oligonucleotide Therapies in 2017.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160.Molecular therapy. Nucleic acids · 2017Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Duchenne muscular dystrophy is one of the most common inherited genetic diseases and is caused by mutations to the DMD gene that encodes the dystrophin protein. Recent advances in genome editing and gene therapy offer hope for the development of potential therapeutics. Truncated versions of the DMD gene can be delivered to the affected tissues with viral vectors and show promising results in a variety of animal models. Genome editing with the CRISPR/Cas9 system has recently been used to restore dystrophin expression by deleting one or more exons of the DMD gene in patient cells and in a mouse model that led to functional improvement of muscle strength. Exon skipping with oligonucleotides has been successful in several animal models and evaluated in multiple clinical trials. Next-generation oligonucleotide formulations offer significant promise to build on these results. All these approaches to restoring dystrophin expression are encouraging, but many hurdles remain. This review summarizes the current state of these technologies and summarizes considerations for their future development.
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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.